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Results for “Spinal Muscular Atrophy 1”

62 trials

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Showing 20 of 62 results

Not applicableApproved For MarketingNCT04256265
What this trial is testing

An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Who this might be right for
Muscular Atrophy, Spinal
Genentech, Inc.
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 applicableLooking for participantsNCT06194539
What this trial is testing

Assessing the Fertility Status of Men With Spinal Muscular Atrophy (SMA)

Who this might be right for
Fertility Issues
Bar-Chama, Natan, M.D. 75
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
Testing effectiveness (Phase 2)Study completedNCT02268552
What this trial is testing

An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)

Who this might be right for
Spinal Muscular Atrophy
Novartis Pharmaceuticals 40
Large-scale testing (Phase 3)Active Not RecruitingNCT04042025
What this trial is testing

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Who this might be right for
Spinal Muscular Atrophy Type ISpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III+1 more
Novartis Gene Therapies 85
Testing effectiveness (Phase 2)Looking for participantsNCT07070999
What this trial is testing

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Who this might be right for
Spinal Muscular Atrophy Type I
Gemma Biotherapeutics 22
Not applicableUnknownNCT02831296
What this trial is testing

Prospective Evaluation of Infants With Spinal Muscular Atrophy:

Who this might be right for
Spinal Muscular Atrophy
Massachusetts General Hospital 1,000
Not applicableEnded earlyNCT01547871
What this trial is testing

Infants With Spinal Muscular Atrophy Type I

Who this might be right for
Spinal Muscular Atrophy
National Institute of Neurological Disorders and Stroke (NINDS) 4
Testing effectiveness (Phase 2)Study completedNCT02913482
What this trial is testing

Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

Who this might be right for
Muscular Atrophy, Spinal
Hoffmann-La Roche 62
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
Testing effectiveness (Phase 2)Study completedNCT03437941
What this trial is testing

Study to Evaluate CORT125281 in Combination With Enzalutamide in Patients With mCRPC

Who this might be right for
Metastatic Castration-Resistant Prostate Cancer
Corcept Therapeutics 39
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
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)Enrolling By InvitationNCT05152823
What this trial is testing

Gene Therapy for IGHMBP2-Related Diseases

Who this might be right for
SMARD1CMT2S
Megan Waldrop 10
Testing effectiveness (Phase 2)Ended earlyNCT00439218
What this trial is testing

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

Who this might be right for
Spinal Muscular Atrophy Type I
Westat 5
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
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 completedNCT01862042
What this trial is testing

Palliative Care in Spinal Muscular Atrophy (SMA) 1

Who this might be right for
Spinal Muscular Atrophy 1
Assistance Publique - Hôpitaux de Paris 39
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
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