Not applicableLooking for participantsNCT04292574What this trial is testingUK SMA Patient RegistryWho this might be right forSpinal Muscular AtrophySMA Newcastle University 800
Testing effectiveness (Phase 2)Study completedNCT00227266What this trial is testingValproic Acid and Carnitine in Patients With Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy University of Utah 94
Not applicableLooking for participantsNCT06562283What this trial is testingEvaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular AtrophyWho this might be right forSpinal AmyotrophyInfantile Spinal Muscular AtrophyJuvenile Spinal Muscular Atrophy Centre Hospitalier Universitaire de Saint Etienne 80
Testing effectiveness (Phase 2)Study completedNCT02908685What this trial is testingInvestigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) ParticipantsWho this might be right forMuscular Atrophy, Spinal Hoffmann-La Roche 231
Not applicableActive Not RecruitingNCT03709784What this trial is testingSpinraza in Adult Spinal Muscular AtrophyWho this might be right forSpinal Muscular AtrophySpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type 3 Washington University School of Medicine 148
Not applicableLooking for participantsNCT06839469What this trial is testingEstablishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular DisordersWho this might be right forSpinal Muscular Atrophy Type 3Duchenne Muscular Dystrophy (DMD) Columbia University 106
Not applicableStudy completedNCT02391831What this trial is testingProspective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular AtrophyWho this might be right forType 2 Spinal Muscular AtrophyType 3 Spinal Muscular Atrophy Institut de Myologie, France 81
Not applicableUnknownNCT04888702What this trial is testingHolter of Movement in Patients with SMA Undergoing Treatment.Who this might be right forSpinal Muscular Atrophy Centre Hospitalier Universitaire de Liege 30
Not applicableStudy completedNCT04907162What this trial is testingMusculoskeletal Nociceptive Pain in Participants With Neuromuscular DisordersWho this might be right forPompe Disease (Late-onset)Myotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy Type 2+4 more LMU Klinikum 82
Testing effectiveness (Phase 2)Looking for participantsNCT07047144What this trial is testingEvaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular AtrophyWho this might be right forSpinal Muscular AtrophySMASpinal Muscular Atrophy Type 2+3 more Scholar Rock, Inc. 52
Testing effectiveness (Phase 2)Study completedNCT03921528What this trial is testingAn Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular AtrophyWho this might be right forSpinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+6 more Scholar Rock, Inc. 58
Testing effectiveness (Phase 2)Ended earlyNCT00439569What this trial is testingClinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or IIIWho this might be right forSpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III Westat 9
Not applicableStudy completedNCT03087526What this trial is testingNIPD on CFTC for Triplet Repeat DiseasesWho this might be right forNon Invasive Prenatal Diagnosis University Hospital, Montpellier 60
Not applicableUnknownNCT06137612What this trial is testingSpinal Cord Gray Matter Imaging in Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy Type IIISpinal Muscular Atrophy Type II University Hospital, Basel, Switzerland 52
Testing effectiveness (Phase 2)Study completedNCT02227823What this trial is testingSafety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3Who this might be right forSpinal Muscular Atrophy Type 3 Centre Hospitalier Régional de la Citadelle 4
Not applicableStudy completedNCT05430113What this trial is testingSpinal Cord Stimulation in Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 4 Marco Capogrosso 3
Testing effectiveness (Phase 2)Looking for participantsNCT06421831What this trial is testingEvaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 PatientsWho this might be right forSpinal Muscular Atrophy Type 3 GeneCradle Inc 21
Testing effectiveness (Phase 2)Study completedNCT01302600What this trial is testingSafety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.Who this might be right forSpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III Non Ambulant Hoffmann-La Roche 165
Not applicableUnknownNCT05475691What this trial is testingLongitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin AmericaWho this might be right forSpinal Muscular Atrophy Hospital Israelita Albert Einstein 361
Large-scale testing (Phase 3)Study completedNCT03837184What this trial is testingSingle-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 CopiesWho this might be right forSpinal Muscular Atrophy Type I Novartis Gene Therapies 2