[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"propionic-acidemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:propionic-acidemia":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,8,0,[8,45,56,83,120,140,162,187],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":24,"conditions":25,"keywords":29,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100054286","natural-history-physiology-microbiome-and-biochemistry-studies-of-propionic-acidemia-100054286",false,"NCT02890342","Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia","The Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia","* INCLUSION CRITERIA:\n* Patients 2 years of age or older, of any gender and ethnicity, with propionic acidemia are eligible to enroll in this protocol. Patients diagnosis will be confirmed based on biochemical and\u002For molecular and enzymatic testing. Participants of any gender and ethnicity over 1 month of age are eligible to enroll remotely for collection of outside records and natural history data. They will be eligible to enroll in the full study for in-person evaluation at 2 years of age.\n* Unaffected family members over 1 month of age, of any ethnicity or race, may be included in the study as household controls for microbiome studies and\u002For for genetic analysis. Studies in unaffected family members may include collection of medical and family history; if necessary completion of physical examination; drawing of blood for research purposes include testing of DNA; collection of stool samples for microbiome studies; collection of dietary history using pen-and-paper or electronic food diary and questionnaires; collection of saliva for metabolite and DNA analysis. In some unaffected family members without a known familial cause of propionic acidemia, exome sequencing or genome sequencing could be performed. Unaffected family members will not receive direct benefit from taking part in the study.\n* If a participant becomes pregnant while on study, the participant can remain on study. The only way to learn more about the critical biological differences in those who affected with propionic acidemia who are pregnant is to continue to follow pregnant women on study.\n\nHowever, no tests or procedures that are greater then minimal risk will be performed. Affected subjects who are pregnant may undergo procedures as part of their clinical care, including blood draws, genetic studies, and consultations, according to the clinical judgement of the clinical team. However, pregnant participants will be excluded from procedures such as organ tissue collection, stable isotope studies, GFR testing, and brain or cardiac MRI until the pregnancy is concluded.\n\n* Healthy volunteers may be eligible to participate in the study if they are between 12 - 40 years of age, must meet specific BMI criteria (similar to affected individuals studied).\n* Patients with propionic acidemia over 1 month of age, of any gender and ethnicity, undergoing a transplantation surgery at Children s Hospital of Pittsburgh, are eligible to participate in the tissue collection arm of the study.\n\nEXCLUSION CRITERIA:\n\n* The PI\u002FAI may decline to enroll a patient because of poor metabolic control, lack of a primary metabolic\u002Fgenetics physician, and intercurrent infection are exclusion criteria for this protocol, the likelihood that an acutely ill or poorly controlled patient will enroll will be minimized.\n* A subset of participants may be enrolled in the tissue collection part of the study only (i.e. if they are too sick to travel). We can may also arrange limited remote consultation with our research team and NIH consultants, the participants referring physician and the participant\u002Ftheir legal guardian through the telephone or an NIH supported telehealth platform for participants who are unable to safely travel to NIH. This would not replace a study visit but would be used when travel isn t possible due to extenuating circumstances (e.g. pandemic). Participants would be encouraged to follow-up for a more thorough in-person evaluation when they are able to travel to NIH.\n* For the healthy volunteers, they will be excluded if they have halitosis, cavities, dental or gingival problems, respiratory diseases (for example, asthma or recent history of COVID19), use tobacco products (for example, cigarette smoking or chewing tobacco), or use electronic nicotine delivery systems (for example, use of e-cigarettes or vaping devices), as this may interfere with accurate measurement of their volatile organic compounds. NIH staff and their family members will be eligible to participate in the healthy volunteer portion of the study.",true,"ALL","1 Month","100 Years",{"count":21,"type":22},1045,"ESTIMATED","OBSERVATIONAL","Background:\n\nPeople s bodies need to break down food into the chemicals. These chemicals are used for energy and growth. Some people cannot process all chemicals very well. Too much of some chemicals can cause diseases. One of these diseases is called propionic acidemia (PA). People with PA can have problems with growth, learning heart, abdomen, and other organs. Researchers want to better understand how these problems happen.\n\nObjective:\n\nTo learn more about propionic acidemia and the genes that might contribute to it.\n\nEligibility:\n\nPeople at least 2 years old with PA who can travel to the clinic\n\nSome unaffected family members\n\nDesign:\n\nParticipants will have a 3 to 5-day hospital visit every year or every few years. Family members may have just 1 visit.\n\nDuring the family member visit, they may have:\n\nMedical history\n\nPhysical exam\n\nSamples of blood and urine\n\nQuestions about diet and a food diary\n\nDoctors and nurses may do additional studies:\n\nSamples of saliva, skin and stool\n\nFluid from a gastronomy tube, if participants have one\n\nDental and eye evaluations\n\nA kidney test - a small amount of dye will be injected and blood will be collected.\n\nConsultations with specialists\n\nA test of calories needed at rest. A clear plastic tent is placed over the participant to measure breathing.\n\nStable isotope study. Participants will take a nonradioactive substance then blow into a bag.\n\nPhotos taken of the face and body with underwear on\n\nUltrasound of the abdomen\n\nHeart tests\n\nHand x-ray\n\nBrain scan\n\nParticipants may have other tests if study doctors recommend them. They will get the results of standard medical tests and genetic tests.",[26,27,28],"Metabolic Disease","Propionic Acidemia","Organic Acidemia",[28,30,31],"Inborn Errors of Metabolism","Natural History","RECRUITING","2026-07-10",{"date":35,"type":36},"2026-07-13","ACTUAL",{"date":38,"type":36},"2016-11-29",{"date":40,"type":22},"2036-08-31",{"name":42,"class":43},"National Human Genome Research Institute (NHGRI)","NIH",2,{"id":46,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":47,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":24,"conditions":48,"keywords":49,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":50,"lastUpdatePostDateStruct":51,"startDateStruct":53,"completionDateStruct":54,"leadSponsor":55,"locationsCount":44},"100277362",{"count":21,"type":22},[26,27,28],[28,30,31],"2026-07-01",{"date":52,"type":36},"2026-07-02",{"date":38,"type":36},{"date":40,"type":22},{"name":42,"class":43},{"id":57,"slug":58,"hasResults":11,"nctId":59,"briefTitle":60,"officialTitle":61,"acronym":4,"eligibilityCriteria":62,"healthyVolunteers":11,"sex":17,"minAge":63,"maxAge":64,"enrollmentInfo":65,"targetDuration":4,"studyType":67,"phases":68,"briefSummary":70,"conditions":71,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":72,"lastUpdatePostDateStruct":73,"startDateStruct":75,"completionDateStruct":77,"leadSponsor":79,"locationsCount":82},"100641521","phase-1-aavrh10-pcca-gene-therapy-for-propionic-acidemia-100641521","NCT07643844","AAVrh10-PCCA Gene Therapy for Propionic Acidemia","Phase 1 Study of Intravenous Administration of a Serotype rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human Propionyl-CoA Carboxylase cDNA (AAVrh10-PCCA) to Individuals With Propionic Acidemia","Inclusion Criteria:\n\n* Age six months to 2 years of age at day of vector infusion. For those \\\u003C1 year of age they must have been ≥37 weeks gestational age at the time of birth and without other conditions\u002Fcomorbidities that in the opinion of the Investigator may interfere with the interpretation of study results.\n* Confirmed diagnosis of propionic acidemia with biallelic PCCA gene mutations based on molecular genetic testing.\n* Study participants must have a diagnosis of neonatal-onset propionic acidemia with a documented episode of decompensation that can include any of the following findings: lethargy, poor feeding, irritability, vomiting, encephalopathy, respiratory failure, seizures, coma, metabolic acidosis, lactic acidosis, ketonuria, hypoglycemia, hyperammonemia, and cytopenias or history of recurrent hospitalizations.\n* Parents or legal guardians of study participants must agree to comply in good faith with the conditions of the study, including attending all of the required baseline and follow-up assessments, and parents or legal guardians must give consent for their child's participation.\n\nExclusion Criteria:\n\n* Hemoglobin \\\u003C10 g\u002Fdl\n* Platelet count \\\u003C 100,000 per mm3\n* Liver Enzyme ALT\u002FAST \\>2.5 ULN\n* Direct Bilirubin \\> 1.5\n* Active viral infection (includes HIV or serology positive for hepatitis B or C).\n* Previous liver transplant\n* Subjects with active decompensation as demonstrated by a pH \\\u003C 7.3, bicarbonate \\\u003C 15 mmol\u002FL, NH3 \\> 75 mcmol\u002FL, lactate \\> 2.5 mmol\u002FL, urine ketones\n* Previously received gene therapy or messenger ribonucleic acid (mRNA) treatments for PA.\n* Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.\n* Family does not want to disclose patient's study participation with primary care physician and other medical providers.","6 Months","2 Years",{"count":66,"type":22},9,"INTERVENTIONAL",[69],"PHASE1","Propionic acidemia is a genetic metabolic disorder characterized by metabolic acidosis, ketosis, vomiting, lethargy, cognitive impairment, and risk of death. It results from loss of function of the mitochondrial enzyme propionyl-CoA carboxylase and can be due to disease-causing variants in the PCCA gene, leading to accumulation of propionyl-CoA and its toxic metabolites. The purpose of this trial is to evaluate the safety and potential therapeutic benefit of an AAV-based gene therapy for propionic acidemia in patients with genetically confirmed biallelic variants in PCCA.",[27],"2026-06-18",{"date":74,"type":36},"2026-06-23",{"date":76,"type":22},"2026-06",{"date":78,"type":22},"2033-12",{"name":80,"class":81},"Mayo Clinic","OTHER",1,{"id":84,"slug":85,"hasResults":11,"nctId":86,"briefTitle":87,"officialTitle":88,"acronym":4,"eligibilityCriteria":89,"healthyVolunteers":11,"sex":17,"minAge":90,"maxAge":4,"enrollmentInfo":91,"targetDuration":4,"studyType":67,"phases":93,"briefSummary":95,"conditions":96,"keywords":97,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":119},"100449281","phase-1-a-study-to-assess-the-long-term-safety-and-clinical-activity-of-mrna-3927-in-participants-previously-enrolled-in-the-mrna-3927-p101-study-100449281","NCT05130437","A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study","A Phase 1\u002F2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study","Inclusion Criteria:\n\n* Participated in Study mRNA-3927-P101.\n* Completed the EOT\u002FET visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in the mRNA-3927-P101 study.\n\nExclusion Criteria:\n\n* Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.\n* Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.\n* History of liver and\u002For kidney transplant.","1 Year",{"count":92,"type":22},50,[69,94],"PHASE2","The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).",[27],[98,99,100,101,102,103,104,105,106,107,108],"mRNA-3927","Propionic Aciduria","Metabolism, Inborn Errors","Genetic Diseases","Inborn Amino Acid Metabolism, Inborn Errors","Acidosis","Acid-Base Imbalance","Metabolic Diseases","Organic Acidemias","Moderna","mRNA","2026-03-20",{"date":111,"type":36},"2026-03-23",{"date":113,"type":36},"2021-11-09",{"date":115,"type":22},"2031-12-04",{"name":117,"class":118},"ModernaTX, Inc.","INDUSTRY",22,{"id":121,"slug":122,"hasResults":11,"nctId":123,"briefTitle":124,"officialTitle":125,"acronym":4,"eligibilityCriteria":126,"healthyVolunteers":11,"sex":17,"minAge":64,"maxAge":4,"enrollmentInfo":127,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":129,"conditions":130,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":131,"lastUpdatePostDateStruct":132,"startDateStruct":134,"completionDateStruct":136,"leadSponsor":138,"locationsCount":139},"100498389","a-retrospective-study-to-characterize-participants-with-propionic-acidemia-100498389","NCT05769621","A Retrospective Study to Characterize Participants With Propionic Acidemia","A Retrospective Cohort Study to Characterize Propionic Acidemia Patients","Inclusion Criteria:\n\nParticipants' medical records are eligible for abstraction only if all of the following criteria apply:\n\n* Confirmed diagnosis of PA based on diagnosis by molecular genetic testing (propionyl-CoA carboxylase subunitα\\[PCCA\\] and\u002For propionyl-CoA carboxylase subunitβ\\[PCCB\\] mutations).\n* Participant provided informed consent (and assent, as applicable).\n* Medical records with sufficient data to support medical encounter for CEC adjudication (MEA adjudication) must be available dating back to birth or 01 January 2015, whichever occurs later. Participants born prior to 2015 will require additional information to confirm eligibility.\n* Experienced at least one MDE in the 24 months preceding the Index Date or experienced at least 3 MDEs within any one 12-month retrospective review period (based on Index Date) dating back to birth or 01 January 2015, whichever occurs later.\n* Must have a confirmed age (months) of disease onset (early or late, where early is defined as the neonatal period).\n* Participants who meet any of the censoring criteria listed below may be enrolled into the study if they have a minimum of 2 years of data available for abstraction prior to meeting the censoring criteria and they meet all inclusion criteria based on the data available for abstraction.\n\nCensoring Criteria:\n\nParticipants medical records meeting the following censoring criterion will not be abstracted:\n\n* Participation in a clinical study of any investigational agent.\n* Received gene therapy treatment\n* Confirmed organ transplantation\n* Investigator is no longer able to obtain relevant clinical information of the participant.\n* Death\n\nExclusion Criteria:\n\nParticipant's medical records will not be eligible for abstraction if the participant meets the following exclusion criteria:\n\n\\- Participation (prior or ongoing) in clinical study mRNA-3927-P101 (NCT04159103) or mRNA-3927-P101-EXT (NCT05130437).",{"count":128,"type":22},60,"This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).",[27],"2026-02-03",{"date":133,"type":36},"2026-02-05",{"date":135,"type":36},"2023-06-16",{"date":137,"type":22},"2026-09-30",{"name":117,"class":118},29,{"id":141,"slug":142,"hasResults":11,"nctId":143,"briefTitle":144,"officialTitle":145,"acronym":4,"eligibilityCriteria":146,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":147,"targetDuration":4,"studyType":67,"phases":149,"briefSummary":150,"conditions":151,"keywords":152,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":153,"lastUpdatePostDateStruct":154,"startDateStruct":156,"completionDateStruct":158,"leadSponsor":160,"locationsCount":161},"100374718","phase-1-open-label-study-of-mrna-3927-in-participants-with-propionic-acidemia-100374718","NCT04159103","Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia","A Global, Phase 1\u002F2, Open-Label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants With Propionic Acidemia","Inclusion Criteria:\n\nParticipants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply:\n\n* ≥ 8 years of age at the time of consent\u002Fassent if enrolled as 1 of the first 2 participants in Part 1.\n* ≥1 year of age at the time of consent\u002Fassent if enrolled after the first 2 participants in Part 1.\n* Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and\u002For PCCB mutations).\n* Part 2 only: At least one documented MDE in the 12-month period before consent.\n\nParticipants \\\u003C1 Year of Age :\n\n* Identification by newborn screening shortly after birth or having suspected PA by presenting with a spectrum of metabolic symptoms, and having a sibling diagnosed with PA. Participant may enter the Screening Period while awaiting genetic testing results, provided that all other eligibility criteria are met but would not be enrolled until diagnosis of PA is confirmed.\n* For infants in the neonatal intensive care unit (NICU) only: ≥37 weeks gestational age at the time of birth without other conditions\u002Fcomorbidities that in the opinion of the Investigator may interfere with the interpretation of study results.\n* Body weight ≥3 kilograms (kg) at Screening.\n* At least 1 documented PA-related event prior to Screening defined as the following criteria:\n\n  * Clinical signs of metabolic deterioration consistent with PA (for example, vomiting, not feeding well\u002Fpoor suck, heavy breathing, lethargy, absence of proper perfusion, abnormal movements including bicycling, abnormal tone, low body temperature, seizure\\[s\\]), OR\n  * Meeting the criteria of MDE definition, OR\n  * Evidence of laboratory abnormalities as evidenced by at least one of the following:\n* Metabolic acidosis with elevated anion gap.\n* Acute hyperammonemia.\n* Neutropenia or thrombocytopenia.\n\nExclusion Criteria:\n\nParticipants of all ages are excluded from the study if during Screening any of the following criteria apply:\n\n* Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in the Investigator or Sponsor's opinion that could interfere with or limit the participation in the study.\n* Estimated glomerular filtration rate (eGFR) \\\u003C30 milliliters (mL)\u002Fminute\u002F1.73 square meter (m\\^2) for participants of all ages receiving chronic dialysis.\n* History of organ transplantation or planned organ transplantation during the period of study participation.\n* Corrected QT interval (QTc) \\>480 milliseconds (ms) using Bazett's correction.\n* Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.\n* Pregnant or breastfeeding.\n* Other clinically significant conditions that in the Investigator's opinion could interfere with the safety of the participant, the interpretation of study results, or limit the participation in the study.",{"count":148,"type":22},77,[69,94],"This 3-part, Phase 1\u002F2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\\\u003C1 year of age).",[27],[98,99,100,101,102,103,104,105,106,107,108],"2026-01-20",{"date":155,"type":36},"2026-01-22",{"date":157,"type":36},"2021-04-15",{"date":159,"type":22},"2027-08-31",{"name":117,"class":118},35,{"id":163,"slug":164,"hasResults":11,"nctId":165,"briefTitle":166,"officialTitle":167,"acronym":4,"eligibilityCriteria":168,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":169,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":171,"conditions":172,"keywords":175,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":177,"lastUpdatePostDateStruct":178,"startDateStruct":180,"completionDateStruct":182,"leadSponsor":184,"locationsCount":186},"100442345","an-observational-study-of-carbaglu-for-the-treatment-of-mma-and-pa-in-adults-and-pediatrics-100442345","NCT05040178","An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics","A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations","Inclusion Criteria:\n\n1. Provision of signed and dated informed consent\u002Fassent form\n2. Prescribed and treated with Carbaglu®\n3. Have an established diagnosis of PA or MMA defined as follows:\n\n   * Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR\n   * Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness).\n\nAND\u002FOR\n\n* Confirmation by molecular genetic testing\n\nExclusion Criteria:\n\n* None",{"count":170,"type":22},20,"To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.",[173,174,27],"Hyperammonemia","Methylmalonic Acidemia",[176],"PA & MMA","2025-04-10",{"date":179,"type":36},"2025-04-15",{"date":181,"type":36},"2022-06-30",{"date":183,"type":22},"2032-06-30",{"name":185,"class":118},"RECORDATI GROUP",5,{"id":188,"slug":189,"hasResults":11,"nctId":190,"briefTitle":191,"officialTitle":191,"acronym":192,"eligibilityCriteria":193,"healthyVolunteers":11,"sex":17,"minAge":63,"maxAge":194,"enrollmentInfo":195,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":197,"conditions":198,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":199,"lastUpdatePostDateStruct":200,"startDateStruct":202,"completionDateStruct":204,"leadSponsor":206,"locationsCount":207},"100376054","understanding-the-long-term-management-of-organic-acidemia-patients-with-carbaglu-a-mixed-methods-approach-100376054","NCT04176523","Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach","PROTECT","Inclusion Criteria:\n\n1. Patient has confirmed diagnosis of an organic acidemia (e.g., MMA or PA)\n2. Patient initiated treatment with carglumic acid for long-term management of MMA or PA\n3. Patient has been treated with carglumic acid for a minimum of 6 months\n4. Patient (or caregiver) is able to comply with all prospective study procedures\n5. Patient (or caregiver) is able to provide informed consent\n\nExclusion Criteria:\n\n* None","99 Years",{"count":196,"type":22},95,"This is a prospective mixed-design study focused on the long-term management of propionic aciduria (PA) and methylmalonic aciduria (MMA) with N-carbamylglutamate (NCG) maintenance therapy. Treatment characteristics, clinical outcomes, and healthcare utilization data of patients diagnosed PA or MMA treated \\>6 months therapy with NCG are collected at baseline, 12 months, 18 months, 36 months and 54 months. Qualitative interviews with adult patients and caregivers are conducted \\>6 months after study enrollment to gain a better understanding of the disease burden and the treatment burden of patients and their families.",[174,27],"2024-02-02",{"date":201,"type":36},"2024-02-05",{"date":203,"type":36},"2019-01-15",{"date":205,"type":22},"2029-07-30",{"name":185,"class":118},32]