[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"early-onset-alzheimer-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:early-onset-alzheimer-disease":58},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,42,71,109,137],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":27,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100457068","phase-1-a-study-to-evaluate-the-safety-and-tolerability-of-aln-app-in-patients-with-eoad-100457068",false,"NCT05231785","A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD","A Randomized, Double-blind, Placebo-controlled Single Ascending Dose and Open-label Multi-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-APP in Adult Patients With Early-onset Alzheimer's Disease (EOAD)","Inclusion Criteria:\n\n* Has mild cognitive impairment or mild dementia due to EOAD\n* Has Clinical Dementia Rating (CDR) global score 0.5 or 1.0 and Mini Mental State Examination (MMSE) \\>20\n\nExclusion Criteria:\n\n* Has Non-Alzheimer's disease dementia\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>2×upper limit of normal (ULN)\n* Has estimated glomerular filtration rate (eGFR) \\\u003C45 mL\u002Fmin\u002F1.73m\\^2 at Screening\n* Has recently received an investigational agent\n* Has recent treatment with amyloid-targeting antibody\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply","ALL","18 Years",{"count":19,"type":20},60,"ESTIMATED","INTERVENTIONAL",[23],"PHASE1","The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple doses of ALN-APP administered by intrathecal (IT) injection in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment duration for Part B: 12 months.",[26],"Early-Onset Alzheimer Disease",[28],"EOAD","RECRUITING","2026-06-10",{"date":32,"type":33},"2026-06-12","ACTUAL",{"date":35,"type":33},"2022-02-04",{"date":37,"type":20},"2029-04-20",{"name":39,"class":40},"Alnylam Pharmaceuticals","INDUSTRY",8,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":48,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":50,"maxAge":4,"enrollmentInfo":51,"targetDuration":4,"studyType":21,"phases":53,"briefSummary":55,"conditions":56,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":70},"100636362","a-study-testing-whether-low-dose-radiation-could-help-the-immune-system-and-possibly-improve-early-onset-alzheimers-disease-100636362","NCT07564700","A Study Testing Whether Low-Dose Radiation Could Help the Immune System and Possibly Improve Early-Onset Alzheimer's Disease.","A Randomized Clinical Feasibility Trial Investigating Low-Dose Radiotherapy as an Immunomodulatory Therapeutic Modality for Early-Onset Alzheimer's Disease: A Pilot Investigation","HOPE","Inclusion Criteria:\n\nAlzheimer's diagnosis according to ICD-10 Ability to understand the clinical study and provide informed consent Early-onset disease Age older than 50 years Stable treatment with anti-dementia medication for at least 3 months\n\nExclusion Criteria:\n\n* Prodromal Alzheimer's disease Mild cognitive impairment (MCI) Severe psychiatric disorders that could interfere with participation in the study Unstable or recently changed medication therapy Other neurodegenerative diseases or severe neurological disorders Uncontrolled chronic diseases that could increase the risk of complications Previous therapeutic brain irradiation Evidence of vascular cognitive impairment on MRI (Fazekas score \\>1 and Wahlund score ≥10\u002F30) Oncological disease (except skin cancer), active or in remission for less than 5 years Evidence of substance abuse (alcohol and\u002For other drugs) with dependence within the last 12 months (DSM-IV criteria) Active or recent (within 3 months) cerebral infection or hemorrhage Immunocompromised status History of seizures Dermatological scalp disease Women who are pregnant, breastfeeding, or planning to become pregnant during the study period Patient has a history of cancer (except non-melanoma skin cancer) Patient is taking antiepileptic medication Patient and legally authorized representative are unable to provide informed consent Patient with a history of focal neurological deficits (except vibratory peripheral neuropathy)","50 Years",{"count":52,"type":20},50,[54],"NA","The goal of this randomized clinical trial is to investigate whether low-dose whole-brain radiotherapy has a potential immunomodulatory effect that may influence disease progression in patients with early-stage Alzheimer's disease (ICD-10 diagnosed).\n\nThe main question(s) it aims to answer are:\n\nDoes low-dose radiotherapy have an effect on cognitive function and disease progression in early Alzheimer's disease? Is the intervention safe and feasible in this patient population under clinical trial conditions?\n\nIf there is a comparison group: Researchers will compare patients receiving low-dose whole-brain radiotherapy to a control group receiving a sham or non-active treatment, to assess potential differences in cognitive outcomes and disease-related parameters over time.\n\nParticipants will:\n\nUndergo baseline diagnostic assessments including neuropsychological testing, MRI, EEG, and laboratory tests Be randomly assigned to one of two study groups Receive either low-dose whole-brain radiotherapy (6 sessions over 3 weeks) or a control condition Attend follow-up visits at 6 weeks, 3 months, and 6 months including repeated cognitive testing and clinical assessments",[57,58],"Alzheimer Disease (AD)","Early Onset Alzheimer Disease","NOT_YET_RECRUITING","2026-04-27",{"date":62,"type":33},"2026-05-04",{"date":64,"type":20},"2026-05-15",{"date":66,"type":20},"2028-05-31",{"name":68,"class":69},"Heinrich-Heine University, Duesseldorf","OTHER",1,{"id":72,"slug":73,"hasResults":11,"nctId":74,"briefTitle":75,"officialTitle":75,"acronym":76,"eligibilityCriteria":77,"healthyVolunteers":78,"sex":16,"minAge":79,"maxAge":80,"enrollmentInfo":81,"targetDuration":4,"studyType":83,"phases":4,"briefSummary":84,"conditions":85,"keywords":88,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":108},"100324688","longitudinal-early-onset-alzheimers-disease-study-protocol-100324688","NCT03507257","Longitudinal Early-onset Alzheimer's Disease Study Protocol","LEADS","Inclusion Criteria for Cognitively Impaired (EOAD and EOnonAD) Cohorts Only:\n\n1. Meets NIA-AA criteria for MCI due to AD or probable AD dementia\n2. Have a global CDR score ≤ 1.0\n3. Have capacity to provide informed consent (IC) or has a legal authorized representative or guardian who provides IC\n4. Age between 40-64 years (inclusive) at the time of consent\n5. Must have a study partner (informant) who spends a minimum average of 10 hours per week with the participant (e.g., family member, significant other, friend, caregiver) who is generally aware of the participants' daily activities and can provide information about the participant's cognitive and functional performance. If the participant does not have a study partner who spends at least 10 face-to-face hours per week, other arrangements for identifying a viable study partner will be granted on a case-by-case basis by the Site PI\n6. Willing and able to complete longitudinal study procedures aside from LP which is an optional procedure\n7. Not pregnant or lactating. Women must be two years post-menopausal, be surgically sterile, or have a negative pregnancy test prior to each PET scan\n8. Fluent in English or Spanish if enrolled in the U.S.\n9. Fluent in English, Spanish, Dutch or Swedish for sites outside the U.S., according to site's spoken language(s).\n\nInclusion Criteria for Cognitively Normal (CN) Cohort Only:\n\n1. Meets criteria for cognitively normal, based on an absence of significant impairment in cognitive functions or activities of daily living\n2. Have a global CDR score = 0\n3. Have capacity to provide informed consent\n4. Have a Mini-Mental State Exam score between 26-30 (inclusive). Exceptions may be made for participant with less than 8 years of education at the discretion of the Site PI\n5. Age between 40-64 years (inclusive) at the time of consent\n6. Must have a study partner (informant) who spends a minimum average of 10 hours per week with the participant (e.g., family member, significant other, friend, caregiver) who is generally aware of the participants' daily activities and can provide information about the participant's cognitive and functional performance. If the participant does not have a study partner who spends 10 face-to-face hours per week, other arrangements for identifying a viable study partner will be granted on a case-by-case basis by the Site PI\n7. Willing and able to complete longitudinal study procedures aside from LP which is an optional procedure\n8. Not pregnant or lactating. Women must be two years post-menopausal, be surgically sterile, or have a negative pregnancy test prior to each PET scan\n9. Fluent in English or Spanish if enrolled in the U.S.\n10. Fluent in English, Spanish, Dutch or Swedish for sites outside the U.S., according to site's spoken language(s).\n\nExclusion Criteria for all (EOAD, EOnonAD and CN) cohorts:\n\n1. Meets core clinical criteria for non-AD dementia\n2. Two or more first degree relatives with a history of early-onset dementia suggestive of autosomal dominant transmission, unless known pathogenic mutations in APP, PSEN1, PSEN2, MAPT, GRN and C9ORF72 have been excluded\n3. Known CLIA certified mutation in an ADAD gene (APP, PSEN1, PSEN2), or other autosomal dominant genes associated with other neurodegenerative disorders (MAPT, GRN, C9ORF72)\n4. Contraindications to 3T MRI (e.g., claustrophobia, pacemaker, select aneurismal clip, artificial heart valve, select ear implants, select stents incompatible with 3T MRI, metal fragments or foreign objects in the eyes, skin or body, etc.)\n5. Lifetime medical history of a brain disorder other than the disorder causing dementia except for headache (exceptions are allowed at the discretion of the Site PI - e.g., seizure disorder thought to be due to EOAD).\n6. MRI scan with evidence of infection or focal lesions, cortical strokes, multiple lacunes (single lacune is allowable unless it meets criteria for strategic lacune affecting cognition)\n7. Any significant systemic illness or unstable medical condition, which could lead to difficulty complying with the protocol (at the discretion of the Site PI)\n8. Research radiation exposure will be assessed by the study physician. If the candidate participant has had more than one nuclear medicine study in the prior 12 months for research-related purposes, study inclusion will require approval from the PET Core\n9. Investigational agents are prohibited 30 days prior to entry\n10. Previous enrollment in a therapeutic trial targeting amyloid or tau.\n11. Participation in other clinical studies with neuropsychological measures, with the exception of participants who are co-enrolled in the NACC Uniform Data Set (UDS) protocol (Note: This criterion is intended to reduce repeat measures effects during neuropsychological testing. Exceptions are allowed at the discretion of the Site PI)\n12. Lifetime history of schizophrenia spectrum disorders (DSM-5 criteria)\n13. Current history (in previous 12 months) of DSM-5 diagnosis of mania, bipolar disorder with or without psychotic features\n14. Current history (in previous 6 months) of moderate or severe substance abuse (nicotine or caffeine is allowed)\n15. Suicidal behaviors in the past 12 months or active suicidal ideations\n16. Residing in a 24-hour care skilled nursing facility (at the time of screening)\n17. (For optional lumbar puncture procedure only):\n\n    a. Clinical laboratory values must be within normal limits or, if abnormal, must be judged to be not clinically significant by the Site PI i. Platelet count \\\u003C100,000\u002Fml ii. INR\\>1.2 iii. Abnormal PT or PTT at screening b. Contraindications to the procedure, including but not limited to severe degenerative joint disease, deformity of the spine, history of a bleeding disorder c. Suspected elevated intracranial pressure, Arnold Chiari malformation or mass lesion d. Use of the anticoagulant medications such as but not limited to warfarin, rivaroxaban, dabigatran\n18. Deemed ineligible by the Site PI for any other reason",true,"40 Years","64 Years",{"count":82,"type":20},850,"OBSERVATIONAL","The Longitudinal Early-onset Alzheimer's Disease Study (LEADS) is a non-randomized, natural history, non-treatment study designed to look at disease progression in individuals with early onset cognitive impairment. Clinical, cognitive, imaging, biomarker, and genetic characteristics will be assessed across three cohorts: (1) early onset Alzheimer's Disease (EOAD) participants, (2) early onset non-Alzheimer's Disease (EOnonAD) participants, and (3) cognitively normal (CN) control participants.",[58,86,87],"Alzheimer Disease","Mild Cognitive Impairment",[89,90,91,92,93,94,95,96,97,98,87],"Cognitively Normal","Amyloid","Plaques","Neuroimaging","Biomarkers","Cognition Disorder","Dementia","Tau","Early Onset Alzheimer's Disease","Alzheimer's Disease","2026-02-17",{"date":101,"type":33},"2026-02-18",{"date":103,"type":33},"2018-04-30",{"date":105,"type":20},"2026-05-31",{"name":107,"class":69},"Indiana University",23,{"id":110,"slug":111,"hasResults":11,"nctId":112,"briefTitle":113,"officialTitle":114,"acronym":4,"eligibilityCriteria":115,"healthyVolunteers":78,"sex":16,"minAge":116,"maxAge":117,"enrollmentInfo":118,"targetDuration":4,"studyType":21,"phases":120,"briefSummary":123,"conditions":124,"keywords":127,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":128,"lastUpdatePostDateStruct":129,"startDateStruct":131,"completionDateStruct":133,"leadSponsor":135,"locationsCount":70},"100592692","phase-2-a-study-of-donanemab-rg6289-or-the-combination-of-donanemab-and-rg6289-in-presenilin-1-psen1-e280a-mutation-carriers-for-the-treatment-of-autosomal-dominant-alzheimers-disease-100592692","NCT06996730","A Study of Donanemab, RG6289, or the Combination of Donanemab and RG6289 in Presenilin 1 (PSEN1) E280A Mutation Carriers for the Treatment of Autosomal-Dominant Alzheimer's Disease","A Double-Blind, Placebo-Controlled, Double-Dummy Study of Donanemab and RG6289 in PSEN1 E280A Mutation Carriers, and in Non-Randomized, Placebo-Treated Non-Carriers From the Same Kindred, to Evaluate the Efficacy and Safety of Donanemab, RG6289, or the Combination of Donanemab and RG6289, in the Treatment of Autosomal-Dominant Alzheimer's Disease","Inclusion Criteria:\n\n* Membership in PSEN1 E280A mutation carrier kindred.\n* Agrees to conditions of, and is willing to undergo, genetic testing (e.g., APOE, PSEN1 E280A, and other genetic testing allowed by local regulatory requirements).\n* Males and females aged 25-65 inclusive.\n* Must meet one of the following criteria:\n\n  a. Determined to be cognitively normal as defined by an MMSE of ≥24 for participants with less than 9 years of education or MMSE of ≥26 for participants with 9 or more years of education. b. Or determined to have MCI with amnestic presentation as defined by:\n\n  1\\. Cognitive concern in the judgment of the Investigator, based in part on the: i. CERAD Word List: Recall \\\u003C3 for participants with less than 9 years of education. ii. CERAD Word List: Recall \\\u003C5 for participants with 9 or more years of education. iii. Preservation of independence in functional activities in the judgment of the Investigator. c. Or determined to have mild AD dementia as defined by:\n  1. Meets the 2011 National Institute on Aging and the Alzheimer's Association (NIA-AA) criteria for probable AD, and\n  2. Has a CDR-GS of 0.5 or 1, with the memory box score ≥0.5.\n\n     Exclusion Criteria:\n* Significant medical, psychiatric, or other neurological condition or disorder documented by history, physical, neurological, laboratory examinations that would place the participant at undue risk in the Investigator's judgment or impact the interpretation of efficacy.\n* History of stroke.\n* History of severe, clinically significant (persistent neurological deficit or structural brain damage) CNS trauma (e.g., cerebral contusion).\n* Current presence of bipolar disorder or other clinically significant major psychiatric disorder according to Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, Text Revision (DSM-5-TR) or symptom (e.g., hallucinations, agitation, paranoia) that could affect the participant's ability to complete evaluations.\n* History of seizures (excluding febrile seizures of childhood, or other isolated seizure episodes that were not due to epilepsy in the judgment of the Investigator, and required at most time-limited anticonvulsant treatment, and which occurred more than 7 years prior to the screening visit).\n* Women who are pregnant or intend to become pregnant during the conduct of this study.\n* Women who are nursing infants or intend to nurse infants during the conduct of this study.\n* Known (or prior) hypersensitivity to donanemab, RG6289, or any excipients of RG6289.\n* History of or active inflammatory bowel disease (e.g., Crohn disease or ulcerative colitis).\n* Medical history of malignancy in the past 5 years, with the following exceptions:\n\n  1. If considered to be cured or,\n  2. If not being actively treated with anti-cancer therapy or radiotherapy and, in the opinion of the Investigator, is not likely to require treatment in the ensuing 5 years and,\n  3. For prostate cancer or basal cell carcinoma, no significant progression over the previous 2 years.\n  4. In-situ cervix carcinoma that has been successfully treated.\n  5. Fully excised non-melanoma skin cancers or in-situ melanoma.\n* Any surgery or hospitalization during the 4 weeks prior to screening or pre-planned\u002Fscheduled during the study period that, in the opinion of the Investigator, may compromise eligibility to the study. The Medical Monitor is available to the Investigator to advise and answer any questions.\n* Inability to tolerate MRI procedures or contraindication to MRI, including, but not limited to, presence of pacemakers not compatible with MRI, aneurysm clips, artificial heart valves, ear implants, or foreign metal objects in the eyes, skin, or body that would contraindicate an MRI scan, or any other clinical history or examination finding that, in the judgment of the Investigator, would pose a potential hazard in combination with MRI\n* Prior participation in an anti-amyloid therapy trial is allowed if it has been at least 1 year since the last study dose.\n* Any other investigational treatment, including anti-amyloid small molecules (i.e., BACEi and GSIs, other GSMs) within five half-lives or 16 weeks prior to screening (calculated from the last safety follow-up visit of the previous study), whichever is longer. Note: The participant may be eligible for this study if it can be documented that he\u002Fshe was randomized to placebo","25 Years","65 Years",{"count":119,"type":20},240,[121,122],"PHASE2","PHASE3","The study will be conducted in 2 blinded parts (Part 1 and Part 2). In Part 1, study participants who are mutation carriers will receive active donanemab and non-mutation carriers will receive placebo-donanemab for up to 18 months (76 weeks), with a minimum treatment period of 9 months. Amyloid PET scans will be conducted at screening, 9, and 18 months in Part 1. Participants who are at or below 11 CL at screening or reach complete amyloid plaque clearance as measured by florbetapir F18 PET (defined as ≤11 CL) at 9 months will initiate Part 2. Participants who are ≤11 CL at screening may delay their entry into Part 2 for up to 6 months at the discretion of the Investigator. All remaining participants will start Part 2 after completing 18 months (76 weeks) in Part 1 independent of amyloid results. Non-carriers will receive placebo in both Parts 1 and 2.\n\nIn Part 2, study participants who are mutation carriers will be randomized 1:1:1:1 in a full factorial design to receive either RG6289 + placebo-donanemab (RG6289 alone group), donanemab + placebo-RG6289 (donanemab alone group), the combination of RG6289 and donanemab (combination group), or placebo-RG6289 and placebo-donanemab (placebo group). All non-carriers will be assigned to the placebo group. CDR-GS at the end of Part 1 and the amyloid level using the last completed amyloid PET scan in Part 1 will be used for stratification. All study participants will participate in a double-dummy design for the duration of Part 2 receiving both an intravenous (IV) infusion at the required interval for the donanemab or matching placebo as well as a daily oral treatment of RG6289 or matching placebo.\n\nAn exploratory outcome of Part 1 is a comparison of the amyloid clearance between this ADAD cohort and historical controls using propensity score matching. The primary outcome in Part 2 is change from the start of Part 2 through the end of Part 2 in brain amyloid load in PSEN1 E280A mutation carriers as measured by amyloid PET imaging. Other endpoints will include fluid and imaging biomarkers and measures of cognition and functioning. The maximum study duration for any individual participant will be 3 years, not including the screening or follow-up periods",[125,58,126],"Autosomal Dominant Alzheimers Disease","Alzheimers Disease",[95],"2025-08-01",{"date":130,"type":33},"2025-08-03",{"date":132,"type":20},"2026-01-15",{"date":134,"type":20},"2030-06-01",{"name":136,"class":69},"Banner Health",{"id":138,"slug":139,"hasResults":11,"nctId":140,"briefTitle":141,"officialTitle":141,"acronym":4,"eligibilityCriteria":142,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":143,"targetDuration":4,"studyType":83,"phases":4,"briefSummary":145,"conditions":146,"keywords":163,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":165,"lastUpdatePostDateStruct":166,"startDateStruct":168,"completionDateStruct":170,"leadSponsor":171,"locationsCount":70},"100557519","flower-following-longitudinal-outcomes-with-epidemiology-for-rare-diseases-100557519","NCT06539169","FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases","Inclusion Criteria:\n\n* Any person with a known or suspected rare disease, defined by their prevalence of fewer than 200,000 individuals nationwide. Diseases include but are not limited to:\n\nAlpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN)\n\n\\- Patients or their legally-authorized representative must be willing and able to provide informed consent (and assent, if applicable). Deceased persons may participate via consent of their legally-authorized representative in accordance with applicable Federal and state laws\n\nExclusion Criteria:\n\n* Patient or LAR is unable to provide informed consent.\n* Patient resides in a country other than the United States and is unable to provide access to medical records.",{"count":144,"type":20},1000,"FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA\u002FRNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic\u002Fdigitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.",[147,148,149,150,151,152,153,26,154,155,156,157,158,159,160,161,162],"Alpha-Thalassemia","Beta-Thalassemia","Amyloidosis","Amyotrophic Lateral Sclerosis","Creutzfeld-Jakob Disease","Cystic Fibrosis","Duchenne Muscular Dystrophy","Ehlers-Danlos Syndrome","Huntington Disease","Gaucher Disease","GM1 Gangliosidosis","Myasthenia Gravis","Pompe Disease","Sickle Cell Disease","Transthyretin Amyloid Cardiomyopathy","Rare Diseases",[164],"rare diseases","2024-11-12",{"date":167,"type":33},"2024-11-14",{"date":169,"type":33},"2024-06-10",{"date":30,"type":20},{"name":172,"class":40},"xCures"]