[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"atypical-hemolytic-uremic-syndrome\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:atypical-hemolytic-uremic-syndrome":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,10,0,[8,54,77,102,124,158,182,209,233,640],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":43,"startDateStruct":46,"completionDateStruct":48,"leadSponsor":50,"locationsCount":53},"100500352","phase-3-evaluate-long-term-safety-tolerability-and-efficacy-of-iptacopan-in-study-participants-with-ahus-100500352",false,"NCT05795140","Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS","A Multi-center, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Iptacopan in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Who Have Completed a Preceding Iptacopan Phase 3 Study in aHUS","Inclusion Criteria:\n\n1. Signed informed consent must be obtained prior to participation in the open label extension study\n2. Willing and able to comply with the study Schedule of Activities\n3. Participants who have completed the full study treatment period of any prior \"Novartis sponsored\" iptacopan Phase 3 clinical trial in aHUS (e.g. CLNP023F12301 , CLNP023F12302), are still on iptacopan study treatment and derive benefit from it as per Investigator's judgement\n4. Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e., any boosters required should be administered according to local guidelines)\n\nExclusion Criteria:\n\n1. Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs\n2. Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the Investigator could put the participant at risk\n3. Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae\n4. History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes\n5. Pregnant or nursing (lactating) women\n6. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after stopping of investigational drug.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.","ALL","18 Years","100 Years",{"count":20,"type":21},65,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.",[27],"Atypical Hemolytic Uremic Syndrome",[29,30,31,32,33,34,35,36,37,38,39,40],"aHUS","iptacopan","atypical hemolytic uremic syndrome","thrombotic microangiopathy","TMA","dialysis","CKD","eGFR","PE\u002FPI","Complement 3 Glomerulopathy (C3G)","IgAN","PNH","RECRUITING","2026-06-01",{"date":44,"type":45},"2026-06-04","ACTUAL",{"date":47,"type":45},"2024-05-08",{"date":49,"type":21},"2032-07-27",{"name":51,"class":52},"Novartis Pharmaceuticals","INDUSTRY",9,{"id":55,"slug":56,"hasResults":11,"nctId":57,"briefTitle":58,"officialTitle":59,"acronym":4,"eligibilityCriteria":60,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":61,"targetDuration":4,"studyType":22,"phases":63,"briefSummary":64,"conditions":65,"keywords":66,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":76},"100511113","phase-3-efficacy-and-safety-of-switching-from-anti-c5-antibody-treatment-to-iptacopan-treatment-in-study-participants-with-atypical-hemolytic-uremic-syndrome-ahus-100511113","NCT05935215","Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)","A Multicenter, Single Arm, Open-label Study to Evaluate Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With aHUS","Inclusion Criteria:\n\n* Male and female adult participants ≥ 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded.\n\n  •. Currently on the recommended (as per label) dosage regimen of anti-C5 antibody treatment, for at least 3 months prior to entering the screening period.\n* In the opinion of the investigator the participant has responded to anti-C5 antibodytreatment prior to screening and has clinical evidence of response (in absence of PE\u002FPI) during the Screening period.\n\nClinical evidence of response to anti-C5 antibody treatment (in absence of PE\u002FPI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as:\n\n1. Hematological normalization in platelet count ≥150 x 10\\^9\u002FL and LDH below upper limit of normal \\[ULN\\], and\n2. Stable kidney function as defined by serum creatinine values within ±15% during the Screening period\n\n   * Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan.\n   * If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations.\n\nExclusion Criteria:\n\n* History of aHUS disease relapse while on anti-C5 antibody treatment.\n* eGFR \\\u003C 30 ml\u002Fmin\u002F1.73m\\^2\n* Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae.\n* Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration.\n* Kidney, bone marrow transplant (BMT)\u002Fhematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation\n* Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study\n* Any medical condition deemed likely to interfere with the patient's participation in the study",{"count":62,"type":21},50,[24],"The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study participants with aHUS.",[27],[27,67,32,33],"aHUS,","2026-04-24",{"date":70,"type":45},"2026-04-29",{"date":72,"type":45},"2024-02-28",{"date":74,"type":21},"2029-07-19",{"name":51,"class":52},31,{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":4,"eligibilityCriteria":83,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":84,"targetDuration":4,"studyType":22,"phases":86,"briefSummary":88,"conditions":89,"keywords":90,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":92,"lastUpdatePostDateStruct":93,"startDateStruct":95,"completionDateStruct":97,"leadSponsor":99,"locationsCount":101},"100616666","phase-4-post-marketing-clinical-study-of-ravulizumab-in-participants-with-clinical-ahus-100616666","NCT07308574","Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS","Multicenter, Open-label, Single-arm, Post-Marketing Clinical Study to Evaluate the Efficacy and Safety of Ravulizumab in Participants Clinically Diagnosed as Atypical Hemolytic Uremic Syndrome","Inclusion Criteria:\n\n* Body weight ≥20 kilograms (kg)\n* Participants clinically diagnosed as aHUS who have any of diseases\u002Fconditions listed below (including participants in whom Thrombotic microangiopathy (TMA) has not been improved even after treatment for the pathogenesis of diagnosed secondary TMA and therefore, diagnosis of aHUS was made).\n* Infection (except for pneumococcal infection and Siga toxin-producing Escherichia coli infection)\n* During pregnancy or postpartum\n* Post-renal transplantation\n* Hypertensive crisis\u002Fmalignant hypertension\n* Systemic lupus erythematosus and related diseases (e.g. dermatomyositis, mixed connective tissue disease, etc.)\n* Participants with the following three signs:\n* Thrombocytopenia: Platelet count \\\u003C150,000\u002Fmicroliter (μL)\n* Microangiopathic haemolytic anaemia: Hb \\\u003C 10 grams per deciliter (g\u002FdL) (\\*)\n* Acute kidney injury: one of the following is fulfilled; 1. ΔsCr ≥ 0.3 milligrams per deciliter (mg\u002FdL) (within 48 hours), 2. 1.5-fold increase from baseline sCr (within 7 days), 3. urinary output ≤ 0.5 mL\u002Fkg\u002Fhour for ≥ 6 hours.\n* No prior treatment with complement inhibitors.\n* The investigator plans to provide the participant with 26-week treatment with ravulizumab in accordance with the treatment policy in clinical practice.\n* Ravulizumab treatment is planned to be initiated within 14 days after onset of the latest TMA episode.\n* Participants consenting to meningococcal vaccine administration and appropriate antibiotic prophylaxis (if required).\n\nExclusion Criteria:\n\n* Participants with TTP, STEC-HUS, secondary TMA that is obviously unrelated to complement abnormality.\n* Participants with TMA caused by malignant tumors, abnormal Cobalamin C metabolism, Streptococcus pneumoniae, drugs, autoimmune diseases other than systemic lupus erythematosus and related diseases (e.g. scleroderma etc.), or hematopoietic stem cell transplantation\n* Participants with pathological complement gene variants (CFH, CFI , CD46 (MCP), C3, CFB, THBD, DGKE) associated with the development of aHUS at enrolment\n* Participants with positive anti-factor H antibodies\n* More than 14 day from onset of TMA to the planned start of ravulizumab treatment\n* Chronic kidney disease or irreversible renal impairment that requires chronic dialysis\n* Presence of unresolved meningococcal disease\n* Judgement by the investigator that the participant is not eligible for the study",{"count":85,"type":21},20,[87],"PHASE4","The primary objective of this study is to assess the platelet count response to ravulizumab in participants clinically diagnosed as atypical hemolytic uremic syndrome (aHUS).",[29,27],[29,31,91],"ravulizumab","2026-04-17",{"date":94,"type":45},"2026-04-20",{"date":96,"type":45},"2025-12-19",{"date":98,"type":21},"2027-06-25",{"name":100,"class":52},"Alexion Pharmaceuticals, Inc.",12,{"id":103,"slug":104,"hasResults":11,"nctId":105,"briefTitle":106,"officialTitle":106,"acronym":107,"eligibilityCriteria":108,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":109,"targetDuration":4,"studyType":111,"phases":4,"briefSummary":112,"conditions":113,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":114,"lastUpdatePostDateStruct":115,"startDateStruct":117,"completionDateStruct":119,"leadSponsor":121,"locationsCount":123},"100609742","the-burden-of-atypical-hemolytic-uremic-syndrome-and-the-clinical-characteristics-of-patients-in-egyptian-hospitals-a-multicenter-observational-retrospective-cohort-study-in-egypt-100609742","NCT07218536","The Burden of Atypical Hemolytic Uremic Syndrome and The Clinical Characteristics of Patients in Egyptian Hospitals A Multicenter, Observational, Retrospective Cohort Study in Egypt","REACH","Inclusion Criteria:\n\n* Male or female patients aged one month or older who have been diagnosed with TMA between 01-Jan-2010 and 31-Dec-2023.\n\nExclusion Criteria:\n\n* None. All records of patients with TMA will be screened for aHUS diagnosis.",{"count":110,"type":21},200,"OBSERVATIONAL","Atypical hemolytic uremic syndrome (aHUS) is a rare, progressive, and life-threatening disease that occurs at any age, with incidence rate of 0.75 to 2.0 cases per million population per year. aHUS is a thrombotic microangiopathy (TMA) commonly caused by dysregulation of the complement system, affecting several organs, especially the kidneys. aHUS can be familial or sporadic, and approximately 50% to 60% of patients have specific identifiable genetic complement mutations and antibodies.\n\nAlthough aHUS is a rare disease, it has a significant impact on the quality of life because of its poor prognosis: a 25% global mortality rate; more than 50% of untreated patients advance to endstage renal disease (ESRD); and more than 75% of adults with renal failure require prompt dialysis. The risk of relapse is also high in many patients, either in the native or transplanted kidneys, so long-term management and close monitoring are essential.\n\nAdvancements in aHUS therapies, especially the availability of anti-complement therapy, have enhanced the natural course of aHUS through hematologic remission induction, kidney function stabilization or improvement, and graft failure prevention. Since complement inhibitors are still unavailable in Egypt, it is important to understand the aHUS manifestations of pediatrics and adults in Egyptian hospitals, aiming for early diagnosis and proper management.\n\nIn this study, we primarily aim to describe the aHUS burden on the patients by gathering their demographic and clinical characteristics, documented disease course, and long-term complications. Our secondary objectives include an estimate of the prevalence of patients diagnosed with aHUS out of all patients with TMA and gathering information about the clinical outcomes of available therapies in real-world settings, as there is no data from the country on aHUS management.",[27],"2026-04-15",{"date":116,"type":45},"2026-04-16",{"date":118,"type":45},"2025-10-13",{"date":120,"type":21},"2026-09-30",{"name":122,"class":52},"AstraZeneca",8,{"id":125,"slug":126,"hasResults":11,"nctId":127,"briefTitle":128,"officialTitle":128,"acronym":129,"eligibilityCriteria":130,"healthyVolunteers":131,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":132,"targetDuration":4,"studyType":22,"phases":134,"briefSummary":136,"conditions":137,"keywords":141,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":147,"lastUpdatePostDateStruct":148,"startDateStruct":150,"completionDateStruct":152,"leadSponsor":154,"locationsCount":157},"100515839","developing-a-pipeline-to-employ-rna-seq-as-a-complementary-diagnostic-tool-in-rare-diseases-100515839","NCT05996731","Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases","ANTHEM","Healthy subjects.\n\nInclusion Criteria:\n\n* Male and female adults\n* Written informed consent\n\nExclusion Criteria:\n\n* Inability to understand the potential risk and benefits of the study\n* Legal incapacity\n\nValidation cohort.\n\nInclusion criteria:\n\n* Male and female adults\n* Genetic diseases affecting RNA levels (frameshifts, stop, large deletions, alteration of canonical splicing sites)\n* Written informed consent\n\nExclusion criteria:\n\n* Underage patients\n* Inability to understand the potential risk and benefits of the study\n* Legal incapacity\n\nDiscovery cohort.\n\nInclusion criteria:\n\n* Male and female patients (children and adults with onset in infancy or early adulthood) with rare genetic undiagnosed diseases\n* Patients with no strong candidates based on previous genetic analysis such as WES, but with clinically suspicion of a genetic rare disease\n* Written informed consent\n\nExclusion criteria:\n\n* Inability to understand the potential risk and benefits of the study\n* Legal incapacity",true,{"count":133,"type":21},105,[135],"NA","This project aims to identify, through RNA-Seq technology, the genetic alterations underlying undiagnosed rare diseases in pediatric and adult patients with early onset and with negative WES.\n\n* Objective 1: Set up and validate techniques. Set-up and validation of the transcriptome analysis protocol in healthy subjects and in patients with known splicing alterations and\u002For altered RNA expression.\n* Objective 2: Diagnostic phase. Study of splicing alterations and RNA levels in cultured fibroblasts obtained from skin biopsies of patients with rare genetic diseases and negative exome.\n\nExploratory goals\n\n* Compare the RNA expression profile obtained from skin biopsy-derived fibroblasts with the RNA expression profile from blood. The most relevant results will be validated in qRT-PCR.\n* To analyze the transcriptional and protein profile heterogeneity in skin-derived fibroblasts in enrolled subjects.\n\nTo explore the effects of genetic (from WES) and transcriptional (from RNA-seq) alterations in participants' plasma and serum.\n\nHealthy controls Five healthy subjects will be recruited from the staff of the Mario Negri Institute for Pharmacological Research. The coded samples will be used to set up the method of isolation and culture of skin fibroblasts and RNA-Seq.\n\nValidation group For the set-up and validation of the skin fibroblast isolation and RNA-Seq procedure, ten adult patients with known diagnosis and with alterations in RNA levels and\u002For splicing will be recruited as positive controls.\n\nPatients who meet the requirements described above will be contacted by the doctors of the Daccò Center for an interview explaining the project. Those who agree to participate in the study will be asked to sign the informed consent before proceeding with the experimental part.\n\n\"Discovery\u002FExploration\" group The exploration cohort will be composed of 30 symptomatic undiagnosed patients with suspected genetic disease (children and adults with infantile onset) belonging to the Clinical Center of the Mario Negri Institute for Pharmacological Research and for whom WES investigations did not reveal causative genetic alterations.",[27,138,139,140],"Membranoproliferative Glomerulonephritis","Autosomal Dominant Polycystic Kidney","Healthy",[142,143,144,145,146],"Undiagnosed genetic rare diseases","Whole-exome sequencing","RNA-Sequencing","Molecular diagnosis","Skin-derived fibroblasts","2026-03-19",{"date":149,"type":45},"2026-03-23",{"date":151,"type":45},"2024-02-21",{"date":153,"type":21},"2026-06",{"name":155,"class":156},"Mario Negri Institute for Pharmacological Research","OTHER",1,{"id":159,"slug":160,"hasResults":11,"nctId":161,"briefTitle":162,"officialTitle":163,"acronym":164,"eligibilityCriteria":165,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":166,"targetDuration":4,"studyType":111,"phases":4,"briefSummary":168,"conditions":169,"keywords":170,"overallStatus":173,"whyStopped":4,"lastUpdateSubmitDate":174,"lastUpdatePostDateStruct":175,"startDateStruct":177,"completionDateStruct":179,"leadSponsor":181,"locationsCount":4},"100623676","ravulizumab-outcomes-in-polish-patients-with-ahus-100623676","NCT07399730","Ravulizumab Outcomes in Polish Patients With aHUS","A Non-interventional Study Evaluating Ravulizumab Treatment Outcomes in Polish Patients With Atypical Hemolytic Uremic Syndrome","aHUS-OPTIMUM","Inclusion Criteria:\n\n* Patients of all ages diagnosed with atypical hemolytic uremic syndrome (aHUS) who received treatment with ravulizumab under the National Drug Program (NDP) in Poland.\n* Patients who are willing to participate in the study and have provided informed consent by signing the informed consent form (ICF).\n\nExclusion Criteria:\n\n* Individuals who intend to participate in a clinical trial for atypical hemolytic uremic syndrome (aHUS) on or after the date of their first ravulizumab infusion through the National Drug Program.\n* Patients with cognitive impairments, those who are unwilling to participate, or those facing language barriers that hinder adequate comprehension or cooperation.",{"count":167,"type":21},80,"This multicenter, observational cohort study uses retrospective collection of past medical history and prospective follow-up to capture longitudinal data on the management and clinical outcomes of patients with atypical hemolytic uremic syndrome (aHUS) treated with ravulizumab as part of routine clinical practice under Poland's National Drug Program (NDP).",[27],[29,27,171,172],"Ravulizumab","Observational","NOT_YET_RECRUITING","2026-02-03",{"date":176,"type":45},"2026-02-10",{"date":178,"type":21},"2026-04-01",{"date":180,"type":21},"2030-12-31",{"name":122,"class":52},{"id":183,"slug":184,"hasResults":11,"nctId":185,"briefTitle":186,"officialTitle":186,"acronym":187,"eligibilityCriteria":188,"healthyVolunteers":131,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":189,"targetDuration":4,"studyType":22,"phases":191,"briefSummary":192,"conditions":193,"keywords":194,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":201,"lastUpdatePostDateStruct":202,"startDateStruct":204,"completionDateStruct":206,"leadSponsor":208,"locationsCount":157},"100501126","functional-implications-of-rare-gene-mutations-in-ahus-open-the-door-to-personalized-therapy-100501126","NCT05805202","Functional Implications of Rare Gene Mutations in aHUS Open the Door to Personalized Therapy","aHUS-iPSC-EC","Inclusion Criteria:\n\n* Adults and children with aHUS defined by history of microangiopathic hemolytic anemia and thrombocytopenia (hematocrit (Ht) \\\u003C30%, hemoglobin (Hb) \\\u003C10 g\u002FdL, LDH \\>500 IU\u002FL, undetectable haptoglobin, fragmented erythrocytes in the peripheral blood smear with negative Coomb's test, and platelet count \\\u003C150,000\u002FmicroL), associated with acute renal failure.\n* Written informed consent\n\nExclusion Criteria:\n\n* TTP (ADAMTS13 activity \\\u003C10%)\n* STEC-HUS (presence of stx and eae genes or Shiga-toxin in the stools and\u002For serum antibodies against Shiga-toxin and\u002For STEC LPS).\n* Disseminated intravascular coagulation (prolonged thromboplastin time and lower than normal fibrinogen levels).",{"count":190,"type":21},112,[135],"Hemolytic Uremic Syndrome (HUS) is a rare disease characterized by rupture of red blood cells (hemolytic anemia), low platelet count (thrombocytopenia), and thrombotic occlusion of small vessels (thrombotic microangiopathy), with prevalent involvement of the kidneys.\n\nSEU, in its typical form is caused by gastrointestinal infection with Escherichia coli.\n\nThe atypical form of SEU (aSEU), which is not caused by an Escherichia coli infection, is a very rare disease that may have a genetic origin; it affects both children and adults and may occur in a sporadic or familial form. Many studies have shown that about 60% of cases of atypical HUS are associated with genetic abnormalities of the complement system (particularly the so-called \"alternative pathway\"), which is a key part of the immune system for responding to infection. Complement consists of a series of proteins that, when activated, create a so-called \"cascade,\" which leads to the elimination of the infectious agent, either directly or through other cells. Complement is finely regulated in such a way as to prevent damage to healthy cells in one's own body. Genetic defects in some of these complement regulatory proteins cause reduced protection of the endothelial surface (thus the vessel wall) against complement activation.\n\nRecently, new mutations have been described in a gene unrelated to the complement pathway, the DKGE gene, which codes for the intracellular isoform of diacylglycerol kinase . In these patients, small renal vessel occlusion appears to occur as a result of altered endothelial cell proliferation and angiogenesis through mechanisms apparently unrelated to complement activation. However, to date these mechanisms are poorly studied. Throughout the entire project statistical methods will be applied to optimize the characterization of the abnormalities in phenotype and function of iPSC-EC derived from aHUS patients with either DGKE or MCP genetic abnormalities as compared with control iPSC-EC, including identifying potential drugs that could correct the abnormalities",[27],[195,196,197,198,199,200],"Atypical hemolytic uremic syndrome","Induced pluripotent stem cells","Endothelial cells","Alternative complement pathway","Eculizumab","Drug screening (in vitro)","2025-09-23",{"date":203,"type":45},"2025-09-29",{"date":205,"type":45},"2023-05-03",{"date":207,"type":21},"2026-12-31",{"name":155,"class":156},{"id":210,"slug":211,"hasResults":11,"nctId":212,"briefTitle":213,"officialTitle":214,"acronym":4,"eligibilityCriteria":215,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":216,"targetDuration":218,"studyType":111,"phases":4,"briefSummary":219,"conditions":220,"keywords":222,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":224,"lastUpdatePostDateStruct":225,"startDateStruct":227,"completionDateStruct":229,"leadSponsor":231,"locationsCount":232},"100172723","atypical-hemolytic-uremic-syndrome-ahus-registry-100172723","NCT01522183","Atypical Hemolytic-Uremic Syndrome (aHUS) Registry","An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)","Inclusion Criteria:\n\n* Male or female patients of any age, including minors, who have been diagnosed with aHUS\n* Patients with or without an identified complement pathogenic variant or anti-complement factor antibody\n* Able to give written informed consent. Patient or patient's parent\u002Flegal guardian must be willing and able to given written informed consent and the patient (if minor) must be willing to give written informed assent \\[if applicable as determined by the central Institutional Review Boards\u002FIndependent Ethics Committees (IRB\u002FIEC)\\].\n* ADAMTS13 \\> 5%, if performed.\n\nExclusion Criteria:\n\n* Hemolytic Uremic Syndrome (HUS) only due to Shiga Toxin producing Escherichia coli (STEC).\n* Unable to give written informed consent. Patient or patient's parent\u002Flegal guardian unable to give written informed consent. Patient (if minor) unable to give written informed assent (if applicable as determined by the central Institutional Review Boards\u002FIndependent Ethics Committees \\[IRB\u002FIEC\\]).",{"count":217,"type":21},3000,"5 Years","Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.",[221],"Atypical Hemolytic-Uremic Syndrome",[221,29,223,33],"Thrombotic Microangiopathy","2025-09-15",{"date":226,"type":45},"2025-09-16",{"date":228,"type":45},"2013-03-18",{"date":230,"type":21},"2031-01-01",{"name":100,"class":52},141,{"id":234,"slug":235,"hasResults":11,"nctId":236,"briefTitle":237,"officialTitle":238,"acronym":239,"eligibilityCriteria":240,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":241,"targetDuration":18,"studyType":111,"phases":4,"briefSummary":243,"conditions":244,"keywords":587,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":630,"lastUpdatePostDateStruct":631,"startDateStruct":633,"completionDateStruct":635,"leadSponsor":637,"locationsCount":639},"100193378","rare-disease-patient-registry--natural-history-study---coordination-of-rare-diseases-at-sanford-100193378","NCT01793168","Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford","Coordination of Rare Diseases at Sanford","CoRDS","Inclusion Criteria:\n\n* Diagnosis of a rare disease, a disease of unknown prevalence, undiagnosed or an unaffected carrier of a rare\u002Funcommon disease\n\nExclusion Criteria:\n\n* Diagnosis of a disease which is not rare",{"count":242,"type":21},20000,"CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org\u002FCoRDS to enroll.",[245,246,247,248,249,250,251,252,253,254,255,256,257,258,259,260,261,262,263,264,265,266,267,268,269,270,271,272,273,274,275,276,277,278,279,280,281,282,283,284,285,286,287,288,289,290,291,292,293,294,295,296,297,298,299,300,301,302,303,304,305,306,307,308,309,310,311,312,313,314,315,316,317,318,319,320,321,322,323,324,325,326,327,328,329,330,331,332,333,334,335,336,337,338,339,340,341,342,343,344,345,346,347,348,349,350,351,352,353,354,355,356,357,358,359,360,361,362,363,364,365,366,367,368,369,370,371,372,373,374,375,376,377,378,379,380,381,382,383,384,385,386,387,388,389,390,391,392,393,394,395,396,397,398,399,400,401,402,403,404,405,406,407,408,409,410,411,412,413,414,415,416,417,418,419,420,421,422,423,424,425,426,427,428,429,430,431,432,433,434,435,436,437,438,439,440,441,442,443,444,445,446,447,448,449,450,451,452,453,454,455,456,457,458,459,460,461,462,463,464,465,466,467,468,469,470,471,472,473,474,475,476,477,27,478,479,480,481,482,483,484,485,486,487,488,489,490,491,492,493,494,495,496,497,498,499,500,501,502,503,504,505,506,507,508,509,510,511,512,513,514,515,516,517,518,519,520,521,522,523,524,525,526,527,528,529,530,531,532,533,534,535,536,537,538,539,540,541,542,543,544,545,546,547,548,549,550,551,552,553,554,555,556,557,558,559,560,561,562,563,564,565,566,567,568,569,570,571,572,573,574,575,576,577,578,579,580,581,582,583,584,585,586],"Rare Disorders","Undiagnosed Disorders","Disorders of Unknown Prevalence","Cornelia De Lange Syndrome","Prenatal Benign Hypophosphatasia","Perinatal Lethal Hypophosphatasia","Odontohypophosphatasia","Adult Hypophosphatasia","Childhood-onset Hypophosphatasia","Infantile Hypophosphatasia","Hypophosphatasia","Kabuki Syndrome","Bohring-Opitz Syndrome","Narcolepsy Without Cataplexy","Narcolepsy-cataplexy","Hypersomnolence Disorder","Idiopathic Hypersomnia Without Long Sleep Time","Idiopathic Hypersomnia With Long Sleep Time","Idiopathic Hypersomnia","Kleine-Levin Syndrome","Kawasaki Disease","Leiomyosarcoma","Leiomyosarcoma of the Corpus Uteri","Leiomyosarcoma of the Cervix Uteri","Leiomyosarcoma of Small Intestine","Acquired Myasthenia Gravis","Addison Disease","Hyperacusis (Hyperacousis)","Juvenile Myasthenia Gravis","Transient Neonatal Myasthenia Gravis","Williams Syndrome","Lyme Disease","Myasthenia Gravis","Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome)","Isolated Klippel-Feil Syndrome","Frasier Syndrome","Denys-Drash Syndrome","Beckwith-Wiedemann Syndrome","Emanuel Syndrome","Isolated Aniridia","Axenfeld-Rieger Syndrome","Aniridia-intellectual Disability Syndrome","Aniridia - Renal Agenesis - Psychomotor Retardation","Aniridia - Ptosis - Intellectual Disability - Familial Obesity","Aniridia - Cerebellar Ataxia - Intellectual Disability","Aniridia - Absent Patella","Aniridia","Peters Anomaly - Cataract","Peters Anomaly","Potocki-Shaffer Syndrome","Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11","Silver-Russell Syndrome Due to Imprinting Defect of 11p15","Silver-Russell Syndrome Due to 11p15 Microduplication","Syndromic Aniridia","WAGR Syndrome","Wolf-Hirschhorn Syndrome","4p16.3 Microduplication Syndrome","4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome","Autosomal Recessive Stickler Syndrome","Stickler Syndrome Type 2","Stickler Syndrome Type 1","Stickler Syndrome","Mucolipidosis Type 4","X-linked Spinocerebellar Ataxia Type 4","X-linked Spinocerebellar Ataxia Type 3","X-linked Intellectual Disability - Ataxia - Apraxia","X-linked Progressive Cerebellar Ataxia","X-linked Non Progressive Cerebellar Ataxia","X-linked Cerebellar Ataxia","Vitamin B12 Deficiency Ataxia","Toxic Exposure Ataxia","Unclassified Autosomal Dominant Spinocerebellar Ataxia","Thyroid Antibody Ataxia","Sporadic Adult-onset Ataxia of Unknown Etiology","Spinocerebellar Ataxia With Oculomotor Anomaly","Spinocerebellar Ataxia With Epilepsy","Spinocerebellar Ataxia With Axonal Neuropathy Type 2","Spinocerebellar Ataxia Type 8","Spinocerebellar Ataxia Type 7","Spinocerebellar Ataxia Type 6","Spinocerebellar Ataxia Type 5","Spinocerebellar Ataxia Type 4","Spinocerebellar Ataxia Type 37","Spinocerebellar Ataxia Type 36","Spinocerebellar Ataxia Type 35","Spinocerebellar Ataxia Type 34","Spinocerebellar Ataxia Type 32","Spinocerebellar Ataxia Type 31","Spinocerebellar Ataxia Type 30","Spinocerebellar Ataxia Type 3","Spinocerebellar Ataxia Type 29","Spinocerebellar Ataxia Type 28","Spinocerebellar Ataxia Type 27","Spinocerebellar Ataxia Type 26","Spinocerebellar Ataxia Type 25","Spinocerebellar Ataxia Type 23","Spinocerebellar Ataxia Type 22","Spinocerebellar Ataxia Type 21","Spinocerebellar Ataxia Type 20","Spinocerebellar Ataxia Type 2","Spinocerebellar Ataxia Type 19\u002F22","Spinocerebellar Ataxia Type 18","Spinocerebellar Ataxia Type 17","Spinocerebellar Ataxia Type 16","Spinocerebellar Ataxia Type 15\u002F16","Spinocerebellar Ataxia Type 14","Spinocerebellar Ataxia Type 13","Spinocerebellar Ataxia Type 12","Spinocerebellar Ataxia Type 11","Spinocerebellar Ataxia Type 10","Spinocerebellar Ataxia Type 1 With Axonal Neuropathy","Spinocerebellar Ataxia Type 1","Spinocerebellar Ataxia - Unknown","Spinocerebellar Ataxia - Dysmorphism","Non Progressive Epilepsy and\u002For Ataxia With Myoclonus as a Major Feature","Spasticity-ataxia-gait Anomalies Syndrome","Spastic Ataxia With Congenital Miosis","Spastic Ataxia - Corneal Dystrophy","Spastic Ataxia","Rare Hereditary Ataxia","Rare Ataxia","Recessive Mitochondrial Ataxia Syndrome","Progressive Epilepsy and\u002For Ataxia With Myoclonus as a Major Feature","Posterior Column Ataxia - Retinitis Pigmentosa","Post-Stroke Ataxia","Post-Head Injury Ataxia","Post Vaccination Ataxia","Polyneuropathy - Hearing Loss - Ataxia - Retinitis Pigmentosa - Cataract","Muscular Atrophy - Ataxia - Retinitis Pigmentosa - Diabetes Mellitus","Non-hereditary Degenerative Ataxia","Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity","Olivopontocerebellar Atrophy - Deafness","NARP Syndrome","Myoclonus - Cerebellar Ataxia - Deafness","Multiple System Atrophy, Parkinsonian Type","Multiple System Atrophy, Cerebellar Type","Multiple System Atrophy","Maternally-inherited Leigh Syndrome","Machado-Joseph Disease Type 3","Machado-Joseph Disease Type 2","Machado-Joseph Disease Type 1","Leigh Syndrome","Late-onset Ataxia With Dementia","Infection or Post Infection Ataxia","GAD Ataxia","Hereditary Episodic Ataxia","Gliadin\u002FGluten Ataxia","Friedreich Ataxia","Fragile X-associated Tremor\u002FAtaxia Syndrome","Familial Paroxysmal Ataxia","Exposure to Medications Ataxia","Episodic Ataxia With Slurred Speech","Episodic Ataxia Unknown Type","Episodic Ataxia Type 7","Episodic Ataxia Type 6","Episodic Ataxia Type 5","Episodic Ataxia Type 4","Episodic Ataxia Type 3","Episodic Ataxia Type 1","Epilepsy and\u002For Ataxia With Myoclonus as Major Feature","Early-onset Spastic Ataxia-neuropathy Syndrome","Early-onset Progressive Neurodegeneration - Blindness - Ataxia - Spasticity","Early-onset Cerebellar Ataxia With Retained Tendon Reflexes","Early-onset Ataxia With Dementia","Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia","Dilated Cardiomyopathy With Ataxia","Cataract - Ataxia - Deafness","Cerebellar Ataxia, Cayman Type","Cerebellar Ataxia With Peripheral Neuropathy","Cerebellar Ataxia - Hypogonadism","Cerebellar Ataxia - Ectodermal Dysplasia","Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss","Brain Tumor Ataxia","Brachydactyly - Nystagmus - Cerebellar Ataxia","Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia","Autosomal Recessive Syndromic Cerebellar Ataxia","Autosomal Recessive Spastic Ataxia With Leukoencephalopathy","Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay","Autosomal Recessive Spastic Ataxia - Optic Atrophy - Dysarthria","Autosomal Recessive Spastic Ataxia","Autosomal Recessive Metabolic Cerebellar Ataxia","Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine","Autosomal Recessive Ataxia, Beauce Type","Autosomal Recessive Ataxia Due to Ubiquinone Deficiency","Autosomal Recessive Ataxia Due to PEX10 Deficiency","Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia","Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency","Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency","Autosomal Recessive Congenital Cerebellar Ataxia","Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome","Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity","Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency","Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect","Autosomal Recessive Cerebellar Ataxia - Saccadic Intrusion","Autosomal Recessive Cerebellar Ataxia - Psychomotor Retardation","Autosomal Recessive Cerebellar Ataxia - Blindness - Deafness","Autosomal Recessive Cerebellar Ataxia","Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly","Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation","Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy","Autosomal Dominant Spastic Ataxia Type 1","Autosomal Dominant Spastic Ataxia","Autosomal Dominant Optic Atrophy","Ataxia-telangiectasia Variant","Ataxia-telangiectasia","Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy","Autosomal Dominant Cerebellar Ataxia Type 4","Autosomal Dominant Cerebellar Ataxia Type 3","Autosomal Dominant Cerebellar Ataxia Type 2","Autosomal Dominant Cerebellar Ataxia Type 1","Autosomal Dominant Cerebellar Ataxia","Ataxia-telangiectasia-like Disorder","Ataxia With Vitamin E Deficiency","Ataxia With Dementia","Ataxia - Oculomotor Apraxia Type 1","Ataxia - Other","Ataxia - Genetic Diagnosis - Unknown","Acquired Ataxia","Adult-onset Autosomal Recessive Cerebellar Ataxia","Alcohol Related Ataxia","Multiple Endocrine Neoplasia","Multiple Endocrine Neoplasia Type II","Multiple Endocrine Neoplasia Type 1","Multiple Endocrine Neoplasia Type 2","Multiple Endocrine Neoplasia, Type IV","Multiple Endocrine Neoplasia, Type 3","Multiple Endocrine Neoplasia (MEN) Syndrome","Multiple Endocrine Neoplasia Type 2B","Multiple Endocrine Neoplasia Type 2A","Atypical HUS","Wiedemann-Steiner Syndrome","Breast Implant-Associated Anaplastic Large Cell Lymphoma","Autoimmune\u002FInflammatory Syndrome Induced by Adjuvants (ASIA)","Hemophagocytic Lymphohistiocytosis","Behcet's Disease","Alagille Syndrome","Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD)","Lowe Syndrome","Pitt Hopkins Syndrome","1p36 Deletion Syndrome","Jansen Type Metaphyseal Chondrodysplasia","Cockayne Syndrome","Chronic Recurrent Multifocal Osteomyelitis","CRMO","Malan Syndrome","Hereditary Sensory and Autonomic Neuropathy Type Ie","VCP Disease","Hypnic Jerking","Sleep Myoclonus","Mollaret Meningitis","Recurrent Viral Meningitis","CRB1","Leber Congenital Amaurosis","Retinitis Pigmentosa","Rare Retinal Disorder","KCNMA1-Channelopathy","Primary Biliary Cirrhosis","ZMYND11","Transient Global Amnesia","Glycogen Storage Disease","Alstrom Syndrome","White Sutton Syndrome","DNM1","EIEE31","Myhre Syndrome","Recurrent Respiratory Papillomatosis","Laryngeal Papillomatosis","Tracheal Papillomatosis","Refsum Disease","Nicolaides Baraitser Syndrome","Leukodystrophy","Tango2","Cauda Equina Syndrome","Rare Gastrointestinal Disorders","Achalasia-Addisonian Syndrome","Achalasia Cardia","Achalasia Icrocephaly Syndrome","Anal Fistula","Congenital Sucrase-Isomaltase Deficiency","Eosinophilic Gastroenteritis","Idiopathic Gastroparesis","Hirschsprung Disease","Rare Inflammatory Bowel Disease","Intestinal Pseudo-Obstruction","Scleroderma","Short Bowel Syndrome","Sacral Agenesis","Sacral Agenesis Syndrome","Caudal Regression","Scheuermann Disease","SMC1A Truncated Mutations (Causing Loss of Gene Function)","Cystinosis","Juvenile Nephropathic Cystinosis","Nephropathic Cystinosis","Kennedy Disease","Spinal Bulbar Muscular Atrophy","Warburg Micro Syndrome","Mucolipidoses","Mitochondrial Diseases","Mitochondrial Aminoacyl-tRNA Synthetases","Mt-aaRS Disorders","Hypertrophic Olivary Degeneration","Non-Ketotic Hyperglycinemia","Fish Odor Syndrome","Halitosis","Isolated Congenital Asplenia","Lambert Eaton (LEMS)","Biliary Atresia","STAG1 Gene Mutation","Coffin Lowry Syndrome","Borjeson-Forssman-Lehman Syndrome","Blau Syndrome","Arginase 1 Deficiency","HSPB8 Myopathy","Beta-Mannosidosis","TBX4 Syndrome","DHDDS Gene Mutations","MAND-MBD5-Associated Neurodevelopmental Disorder","Constitutional Mismatch Repair Deficiency (CMMRD)","SPATA5 Disorder","SPATA5L1 Related Disorder","Acrodysostosis","Multi-systematic Smooth Muscle Dysfunction Syndrome","CRELD1 (Cysteine Rich With EGF Like Domains 1)","GNB1 Syndrome","Pyruvate Dehydrogenase Complex Deficiency Disease","Beta Mannosidosis","Kbg Syndrome","Labrune Syndrome","Metachromatic Leukodystrophy (MLD)","Moyamoya Disease","OPHN1 Syndrome","Oculopharyngeal Muscular Dystrophy (OPMD)","TUBB3 Mutation","WOREE (WWOX-related Epileptic Encephalopathy","SCAR12","Skraban-Deardorff Syndrome","Hereditary Myopathy With Early Respiratory Failure",[588,589,590,591,592,299,593,594,306,595,265,596,597,598,469,27,599,600,256,601,602,264,603,266,604,255,605,606,480,607,608,483,484,609,486,487,610,611,490,612,613,614,540,615,616,617,618,619,620,621,545,622,623,624,550,551,625,626,627,628,629],"Rare Diseases","Neglected Diseases","Orphan Diseases","Rare Disease Research","Registries","Ataxia","Cornelia de Lange Syndrome","Ataxia Telangiectasia","Batten Disease","Mucolipidosis IV","Klippel-Feil Syndrome","Undiagnosed","Uncommon Disease","Hypersomnia","Hyperacusis","Marinesco-Sjogren Syndrome","4p-\u002FWolf-Hirschhorn Syndrome","Narcolepsy","Wiedermann-Steiner Syndrome","Autoimmune\u002Finflammatory Syndrome Induced by Adjuvants (ASIA)","Hemophagocytic Lymphohistiocytosis (HLH)","Inclusion body myopathy with early-onset Paget disease and frontotemporal dementia (IBMPFD)","1p36 deletion syndrome","Jansen metaphyseal chondrodysplasia","Chronic recurrent multifocal osteomyelitis (CRMO)","Malan syndrome","Hereditary Sensory and Autonomic Neuropathy","Juvenile nephropathic cystinosis","Nephropathic infantile cystinosis","Ocular cystinosis","Kennedy disease","Spinal Bulbar Muscular Atrophy (SBMA)","SMC1A Truncated Mutations (causing loss of gene function)","Leigh syndrome","Mucolipidosis","Mitochondrial aminoacyl-tRNA synthetases (Mt-aaRS Disorders)","Shine Syndrome","Intestinal Bromhidrosis Syndrome","Fish odor syndrome","Autosomal recessive extra oral halitosis","CACNA1H mutation","Dimethylglycine dehydrogenase deficiency","2025-05-22",{"date":632,"type":45},"2025-05-29",{"date":634,"type":45},"2010-07",{"date":636,"type":21},"2100-12",{"name":638,"class":156},"Sanford Health",2,{"id":641,"slug":642,"hasResults":11,"nctId":643,"briefTitle":644,"officialTitle":645,"acronym":646,"eligibilityCriteria":647,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":648,"targetDuration":650,"studyType":111,"phases":4,"briefSummary":651,"conditions":652,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":732,"lastUpdatePostDateStruct":733,"startDateStruct":735,"completionDateStruct":737,"leadSponsor":739,"locationsCount":157},"100521150","national-registry-of-rare-kidney-diseases-100521150","NCT06065852","National Registry of Rare Kidney Diseases","National Registry of Rare Kidney Diseases (RaDaR)","RaDaR","* Kidney Rare Disease\n* Paeds and adults\n* Eligibility differs for each rare disease group\n* See: https:\u002F\u002Fukkidney.org\u002Frare-renal\u002Frecruitment",{"count":649,"type":21},35000,"30 Years","The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.\n\nThe purpose of this research is to:\n\n* Develop Clinical Guidelines for specific rare kidney diseases. These are written recommendations on how to diagnose and treat a medical condition.\n* Audit treatments and outcomes. An audit makes checks to see if what should be done is being done and asks if it could be done better.\n* Further the development of future treatments.\n\nParticipants will be invited to participate on clinical trials and other studies. The registry has the capacity to feedback relevant information to patients and in conjunction with Patient Knows Best (Home - Patients Know Best), allows patients to provide information themselves, including their own reported quality of life and outcome measures.",[653,654,655,656,657,27,658,659,660,661,662,663,664,665,666,667,668,669,540,670,671,672,281,673,674,675,676,677,678,679,680,681,682,683,684,685,686,687,688,689,690,691,692,693,694,695,696,697,698,699,700,701,702,703,704,705,486,706,138,707,708,709,710,711,712,713,714,715,716,717,718,719,720,721,722,723,724,725,726,727,728,729,730,731],"Adenine Phosphoribosyltransferase Deficiency","AH Amyloidosis","AHL Amyloidosis","AL Amyloidosis","Alport Syndrome","Autoimmune Distal Renal Tubular Acidosis","Autosomal Recessive Proximal Renal Tubular Acidosis","Autosomal Recessive Distal Renal Tubular Acidosis","Autosomal Dominant Polycystic Kidney Disease","Autosomal Recessive Polycystic Kidney Disease","Bartter Syndrome","BK Nephropathy","C3 Glomerulopathy With Monoclonal Gammopathy","C3 Glomerulopathy","Calciphylaxis","Crystalglobulinaemia","Crystal-storing Histiocytosis","Cystinuria","Dense Deposit Disease","Dent Disease","Dominant Hypophosphataemia With Nephrolithiasis and\u002For Osteoporosis","Drug Induced Fanconi Syndrome","Drug-Induced Hypomagnesemia","Drug-Induced Nephrogenic Diabetes Insipidus","Epilepsy, Ataxia, Sensorineural Deafness and Tubulopathy","Fabry Disease","Familial Hypomagnesemia With Hypercalciuria and Nephrocalcinosis","Familial Primary Hypomagnesemia With Hypocalcuria","Familial Primary Hypomagnesaemia With Normocalciuria","Familial Renal Glucosuria","Fanconi Renotubular Syndrome 1","Fanconi Renotubular Syndrome 2","Fanconi Renotubular Syndrome 3","Fibrillary Glomerulonephritis","Fibromuscular Dysplasia","Focal Segmental Glomerulosclerosis","Generalised Pseudohypoaldosteronism Type 1","Gitelman Syndrome","Heavy-Metal-Induced Fanconi Syndrome","Hepatocyte Nuclear Factor 1-Beta-Associated Monogenic Diabetes","Hereditary Renal Hypouricemia","Hereditary Hypophosphatemic Rickets With Hypercalciuria","Hyperuricaemic Nephropathy","IgA Nephropathy","Immunotactoid Glomerulonephritis With Organised Microtubular Mononoclonal Immunoglobulin Deposits","Inherited Renal Cancer Syndromes","Intracapillary Monoclonal IgM Without Cryoglobulin","Intraglomerular\u002FCapillary Lymphoma\u002FLeukaemia","Isolated Autosomal Dominant Hypomagnesaemia Glaudemans Type","Liddle Syndrome","Light Chain Cast Nephropathy","Light Chain Proximal Tubulopathy Without Crystals","Light Chain Proximal Tubulopathy With Crystals","Membranous Nephropathy","Medullary Cystic Kidney Disease","Minimal Change Nephropathy","Mitochondrial Disease Of The Kidney","Monoclonal Immunoglobulin Deposition Disease","Nail Patella Syndrome","Nephrogenic Diabetes Insipidus","Nephrogenic Syndrome of Inappropriate Antidiuresis","Nephronophthisis","Primary Hypomagnesemia With Secondary Hypocalcemia","Primary Hyperoxaluria","Proliferative Glomerulonephritis With Monoclonal IgG Deposits","Proximal Tubulopathy Without Crystals","Pseudohypoaldosteronism Type 1, 2A-2E","Pure Red Cell Aplasia","Retroperitoneal Fibrosis","Sickle Cell Nephropathy","Shiga Toxin Associated Haemolytic Uraemic Syndrome","Steroid Resistant Nephrotic Syndrome","Steroid-Sensitive Nephrotic Syndrome","Thin Basement Membrane Nephropathy","Thrombotic Microangiopathy With Monoclonal Gammopathy","Type 1 Cryoglobulinaemic Glomerulonephritis","Tuberous Sclerosis","Unclassified Monoclonal Gammopathy Of Renal Significance","Vasculitis","2023-09-26",{"date":734,"type":45},"2023-10-04",{"date":736,"type":45},"2009-11-06",{"date":738,"type":21},"2039-12-31",{"name":740,"class":156},"UK Kidney Association"]