Testing effectiveness (Phase 2)Enrolling By InvitationNCT05152823What this trial is testingGene Therapy for IGHMBP2-Related DiseasesWho this might be right forSMARD1CMT2S Megan Waldrop 10
Not applicableNot Yet RecruitingNCT07838779What this trial is testingAn OLIP Study (Open Label Individual Patient) of Nusinersen in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With NusinersenWho this might be right forSMA - Spinal Muscular Atrophy London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's 1
Not applicableStudy completedNCT00466349What this trial is testingInternational SMA Patient RegistryWho this might be right forMuscular Atrophy, Spinal Indiana University 3,000
Not applicableEnded earlyNCT02550691What this trial is testingIdentification of a Biomarker Associated With Cis-duplication of the SMN1 GeneWho this might be right forSpinal Muscular Atrophy University Hospital, Rouen 48
Not applicableStudy completedNCT01910168What this trial is testingCollect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker AnalysisWho this might be right forMuscular Atrophy, Spinal Hoffmann-La Roche 36
Early research (Phase 1)Study completedNCT02122952What this trial is testingGene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1Who this might be right forSpinal Muscular Atrophy 1 Novartis Gene Therapies 15
Not applicableStudy completedNCT07378943What this trial is testingDosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying TherapiesWho this might be right forMuscular Atrophy, Spinal Novartis Pharmaceuticals 4,114
Not applicableActive Not RecruitingNCT07443449What this trial is testingInternational SBMA Project (KDA)Who this might be right forSBMASpinal and Bulbar Muscular Atrophy (SBMA) Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 700
Not applicableNot Yet RecruitingNCT07567378What this trial is testingCARTIZ Registry: Cartilage, Arthropathy and Imaging Under Tirzepatide in Zone-stratified Cohorts - A Four-Institute Mexican Observational RegistryWho this might be right forPsoriatic ArthritisOsteoarthitisKnee+12 more JULIO GRANADOS MONTIEL 30
Not applicableLooking for participantsNCT07805278What this trial is testingA Registry of Cases of Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy St. Jude Children's Research Hospital 30
Post-approval studies (Phase 4)Study completedNCT04488133What this trial is testingLearn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)Who this might be right forMuscular Atrophy, Spinal Biogen 46
Not applicableUnknownNCT04644393What this trial is testingResponsiveness and Validation Study of MFM-20 in SMA Patients Treated With NusinersenWho this might be right forSpinal Muscular Atrophy Hospices Civils de Lyon 40
Not applicableLooking for participantsNCT07766980What this trial is testingEvaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy PatientsWho this might be right forDuchenne Muscular DystrophyDuchenne DiseaseMuscular Dystrophy in Children+2 more University of Florida 20
Not applicableUnknownNCT02831296What this trial is testingProspective Evaluation of Infants With Spinal Muscular Atrophy:Who this might be right forSpinal Muscular Atrophy Massachusetts General Hospital 1,000
Not applicableEnrolling By InvitationNCT07072676What this trial is testingThe Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Who this might be right forInclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more LMU Klinikum 30
Not applicableStudy completedNCT04004390What this trial is testingTreatment of Post-poliomyelitis Syndrome by Intravenous ImmunoglobulinWho this might be right forPost-Poliomyélitis Syndrom University Hospital, Montpellier 17
Not applicableUnknownNCT05521217What this trial is testingMyotonometer Intra- and Inter Examiner Reliability on Spinal Muscular AtrophyWho this might be right forMuscle Tone PoorSpinal Muscular Atrophy Istanbul Medipol University Hospital 30
Not applicableStudy completedNCT00160082What this trial is testingEfficacy and Safety of Xepol (Human Immunoglobulin) in Subjects With Post-Polio Syndrome (PPS)Who this might be right forPost Polio Syndrome, PPS Calliditas Therapeutics AB 124
Not applicableStudy completedNCT02574390What this trial is testingAnswer ALS: Individualized Initiative for ALS DiscoveryWho this might be right forAmyotrophic Lateral SclerosisPrimary Lateral SclerosisFlail Arm ALS+5 more Johns Hopkins University 1,049
Large-scale testing (Phase 3)Ended earlyNCT00839033What this trial is testingEvaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular DisordersWho this might be right forDuchenne Muscular DystrophyAmyotrophic Lateral SclerosisNeuromuscular Diseases Assistance Publique - Hôpitaux de Paris 14