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Results for “Spinal Muscular Atrophy Type I”

47 trials

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Showing 20 of 47 results

Testing effectiveness (Phase 2)Ended earlyNCT00439569
What this trial is testing

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

Who this might be right for
Spinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III
Westat 9
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 applicableActive Not RecruitingNCT05475691
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 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 applicableEnrolling By InvitationNCT06256887
What this trial is testing

Sleep Spindles Organization as an Early Neural Marker of Neuromotor Outcome

Who this might be right for
EEG Sleep SpindlesMotor OutcomeCerebral Palsy+1 more
IRCCS Fondazione Stella Maris 80
Not applicableStudy completedNCT00466349
What this trial is testing

International SMA Patient Registry

Who this might be right for
Muscular Atrophy, Spinal
Indiana University School of Medicine 3,000
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
Hangzhou Jiayin Biotech Ltd 18
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 applicableStudy completedNCT04193228
What this trial is testing

Muscle Structure, Function and Gait in dHMN

Who this might be right for
Distal Hereditary Motor Neuropathy, Type IIDistal Hereditary Motor Neuropathy, Type VDistal Hereditary Motor Neuronopathy Type I+1 more
University College London Hospitals 11
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 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
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 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
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
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
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
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
Hangzhou Jiayin Biotech Ltd 12
Not applicableActive Not RecruitingNCT03421977
What this trial is testing

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

Who this might be right for
Spinal Muscular Atrophy 1
Novartis Gene Therapies 13
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
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
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