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Results for “Type I Spinal Muscular Atrophy”

49 trials

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Showing 20 of 49 results

Testing effectiveness (Phase 2)Enrolling By InvitationNCT05614531
What this trial is testing

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

Who this might be right for
Spinal Muscular Atrophy Type I
Guangzhou Jiayin Biotech Ltd 12
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
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 applicableUnknownNCT05475691
What this trial is testing

Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America

Who this might be right for
Spinal Muscular Atrophy
Hospital Israelita Albert Einstein 361
Not applicableStudy completedNCT05715749
What this trial is testing

Body Weight Support Harness System in Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy Type ISpinal Muscular Atrophy Type II
Nationwide Children's Hospital 33
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 applicableNot Yet RecruitingNCT07208903
What this trial is testing

Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions

Who this might be right for
Spinal Muscular Atrophy (SMA)Spinal Muscular Atrophy Type I
University Hospital, Strasbourg, France 36
Not applicableStudy completedNCT00756821
What this trial is testing

A Pilot Study of Biomarkers for Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy
Carelon Research 130
Not applicableStudy completedNCT04907162
What this trial is testing

Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

Who this might be right for
Pompe Disease (Late-onset)Myotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy Type 2+4 more
LMU Klinikum 82
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)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
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
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
Testing effectiveness (Phase 2)Study completedNCT00568698
What this trial is testing

A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

Who this might be right for
Muscular Atrophy, Spinal
Stanford University 29
Not applicableNot Yet RecruitingNCT07531719
What this trial is testing

Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)

Who this might be right for
Spinal Muscle Atrophy
Hoffmann-La Roche 30
Not applicableLooking for participantsNCT04292574
What this trial is testing

UK SMA Patient Registry

Who this might be right for
Spinal Muscular AtrophySMA
Newcastle University 800
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 applicableActive Not RecruitingNCT07617779
What this trial is testing

The Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy (SMA)
Nikegen Pharmaceutical (Hangzhou) Company Limited 21
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
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
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