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Results for “Spinal Muscular Atrophy 1”

65 trials

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Showing 20 of 65 results

Not applicableWithdrawnNCT01459302
What this trial is testing

Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders

Who this might be right for
Amyotrophic Lateral SclerosisFrontotemporal DementiaPLS+7 more
University of Massachusetts, Worcester
Testing effectiveness (Phase 2)Looking for participantsNCT05824169
What this trial is testing

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Who this might be right for
Spinal Muscular Atrophy
GeneCradle Inc 18
Testing effectiveness (Phase 2)Study completedNCT05517603
What this trial is testing

Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

Who this might be right for
Spinal and Bulbar Muscular AtrophyKennedy's Disease
AnnJi Pharmaceutical Co., Ltd. 25
Early research (Phase 1)Ended earlyNCT05673109
What this trial is testing

AC176 for the Treatment of Metastatic Castration Resistant Prostate Cancer

Who this might be right for
Metastatic Castration Resistant Prostate Cancer
Accutar Biotechnology Inc 8
Not applicableStudy completedNCT02466529
What this trial is testing

Natural History of Spinal Muscular Atrophy Type 1 in Taiwan

Who this might be right for
Natural History of Type 1 Spinal Muscular Atrophy (SMA)
Kaohsiung Medical University Chung-Ho Memorial Hospital 111
Not applicableNot Yet RecruitingNCT07596277
What this trial is testing

Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication

Who this might be right for
Spinal Muscular Atrophy 1
Guy's and St Thomas' NHS Foundation Trust 15
Not applicableStudy completedNCT02532244
What this trial is testing

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

Who this might be right for
Spinal Muscular AtrophyCharcot-Marie-Tooth DiseaseMuscular Dystrophy+5 more
Nemours Children's Clinic 230
Testing effectiveness (Phase 2)Study completedNCT00661453
What this trial is testing

CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

Who this might be right for
Spinal Muscular Atrophy Type I
University of Utah 40
Not applicableNo Longer AvailableNCT02865109
What this trial is testing

Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

Who this might be right for
Infantile-onset Spinal Muscular Atrophy
Biogen
Testing effectiveness (Phase 2)Enrolling By InvitationNCT05152823
What this trial is testing

Gene Therapy for IGHMBP2-Related Diseases

Who this might be right for
SMARD1CMT2S
Megan Waldrop 10
Not applicableStudy completedNCT03395795
What this trial is testing

Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II

Who this might be right for
Infantile Spinal Muscular Atrophy
Assistance Publique - Hôpitaux de Paris 1
Not applicableStudy completedNCT06632730
What this trial is testing

Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain

Who this might be right for
Spinal Muscular Atrophy Type ISpinal Muscular Atrophy Type II
Novartis 69
Not applicableStudy completedNCT02466217
What this trial is testing

Phenomics in Autoimmune and Inflammatory Diseases

Who this might be right for
Healthy VolunteerRheumatoid ArthritisAnkylosing Spondylitis+10 more
Assistance Publique - Hôpitaux de Paris 537
Testing effectiveness (Phase 2)UnknownNCT02855112
What this trial is testing

Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients

Who this might be right for
Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]
Tehran University of Medical Sciences 10
Large-scale testing (Phase 3)Study completedNCT03306277
What this trial is testing

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Who this might be right for
SMA - Spinal Muscular AtrophyGene Therapy
Novartis Gene Therapies 22
Not applicableApproved For MarketingNCT03955679
What this trial is testing

AveXis Managed Access Program Cohort for Access to AVXS-101

Who this might be right for
Spinal Muscular Atrophy
United BioSource, LLC
Large-scale testing (Phase 3)Study completedNCT03505099
What this trial is testing

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

Who this might be right for
Spinal Muscular Atrophy
Novartis Gene Therapies 30
Not applicableLooking for participantsNCT06562283
What this trial is testing

Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy

Who this might be right for
Spinal AmyotrophyInfantile Spinal Muscular AtrophyJuvenile Spinal Muscular Atrophy
Centre Hospitalier Universitaire de Saint Etienne 80
Not applicableEnrolling By InvitationNCT06588803
What this trial is testing

A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA

Who this might be right for
Spinal Muscular Atrophy
Guangzhou Jiayin Biotech Ltd 18
Not applicableStudy completedNCT05878418
What this trial is testing

The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy Type IScoliosisChest Deformities+3 more
Medipol University 20
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