Not applicableStudy completedNCT03650569What this trial is testingItalian Angelman Syndrome RegistryWho this might be right forAngelman Syndrome FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS 82
Large-scale testing (Phase 3)Study completedNCT05156320What this trial is testingEfficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or RisdiplamWho this might be right forSpinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+7 more Scholar Rock, Inc. 188
Large-scale testing (Phase 3)Study completedNCT00478647What this trial is testingStudy of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With ImigluceraseWho this might be right forGaucher Disease Shire 40
Not applicableStudy completedNCT04805385What this trial is testingEffects of Lactobacillus Plantarum PS128 in Patients With Tourette's SyndromeWho this might be right forTourette's Syndrome China Medical University Hospital 121
Not applicableStudy completedNCT01313325What this trial is testingHippotherapy to Improve the Balance of Children With Movement DisordersWho this might be right forNeuromuscular DiseasesCerebral PalsyDown Syndrome+2 more Central Michigan University 16
Not applicableLooking for participantsNCT03564561What this trial is testingNatural History of Pompe DiseaseWho this might be right forGlycogen Storage Disease Type II, Adult Assistance Publique - Hôpitaux de Paris 20
Not applicableUnknownNCT02000310What this trial is testingMolecular and Cellular Mechanisms of Lysosomal Storage DiseasesWho this might be right forLysosomal Storage Disorders O & O Alpan LLC 80
Not applicableEnded earlyNCT04008849What this trial is testingA Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456Who this might be right forInherited Metabolic Disorders (IMD) Magenta Therapeutics, Inc. 3
Not applicableStudy completedNCT00597948What this trial is testingHealthy Lifestyles for People With Intellectual DisabilitiesWho this might be right forMental RetardationDevelopmental DisabilitiesIntellectual Disabilities Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 131
Not applicableUnknownNCT05558774What this trial is testingLong-term Outcomes After Percutaneous Closure of PFOWho this might be right forForamen Ovale, PatentEchocardiographyEmbolic Stroke+1 more MC Medicor 300
Early research (Phase 1)WithdrawnNCT01130623What this trial is testingA Phase I Study of Pazopanib as a Single Agent for Children With Refractory Solid TumorsWho this might be right forSarcomaNeuroblastomaWilms Tumor+2 more National Cancer Institute (NCI)
Not applicableStudy completedNCT02990676What this trial is testingExploring Computerised Cognitive Training for People With Huntington's DiseaseWho this might be right forHuntington's Disease Cardiff University 30
Not applicableActive Not RecruitingNCT04848779What this trial is testingA Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)Who this might be right forGlycogen Storage Disease Type II Sanofi 16
Testing effectiveness (Phase 2)Looking for participantsNCT05104983What this trial is testingStopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention StudyWho this might be right forTuberous Sclerosis ComplexEpilepsy Darcy Krueger 64
Testing effectiveness (Phase 2)WithdrawnNCT02715505What this trial is testingSafety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)Who this might be right forInherited Metabolic Disorders IMD Novartis Pharmaceuticals
Testing effectiveness (Phase 2)Active Not RecruitingNCT04584983What this trial is testingBilirubin Neurotoxicity (BN) and Neurodevelopmental Impairment (NDI) in Extremely Preterm (EP) Infants: Avoidable by Reducing the Usual Intravenous Lipid (UL) AdministrationWho this might be right forBilirubin Encephalopathy The University of Texas Health Science Center, Houston 134
Not applicableStudy completedNCT04804566What this trial is testingUnderstanding Fabry Disease Therapy Choices Through the Eyes of the PatientsWho this might be right forFabry Disease Amicus Therapeutics 44
Not applicableUnknownNCT04476550What this trial is testingClinical Specimen Collection From Pompe Disease PatientsWho this might be right forPompe Disease Serhat Gumrukcu, MD PhD 12
Not applicableApproved For MarketingNCT00484991What this trial is testingSapropterin Expanded Access ProgramWho this might be right forPhenylketonuria BioMarin Pharmaceutical
Early research (Phase 1)Looking for participantsNCT07246941What this trial is testingEvaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's DiseaseWho this might be right forHuntington's Disease Hoffmann-La Roche 40