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Results for “Spinal Muscular Atrophy Type I”

51 trials

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Showing 20 of 51 results

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 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
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
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 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
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 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 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
Early research (Phase 1)Study completedNCT02122952
What this trial is testing

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

Who this might be right for
Spinal Muscular Atrophy 1
Novartis Gene Therapies 15
Not applicableStudy completedNCT00466349
What this trial is testing

International SMA Patient Registry

Who this might be right for
Muscular Atrophy, Spinal
Indiana University 3,000
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)Looking for participantsNCT07554924
What this trial is testing

A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

Who this might be right for
Spinal Muscular Atrophy 1
Lanyue Biotech (Hangzhou) Co., Ltd. 11
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