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Results for “Infantile Spinal Muscular Atrophy”

35 trials

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Showing 20 of 35 results

Large-scale testing (Phase 3)Study completedNCT03837184
What this trial is testing

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

Who this might be right for
Spinal Muscular Atrophy Type I
Novartis Gene Therapies 2
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
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 completedNCT02061189
What this trial is testing

Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)

Who this might be right for
Infantile Spinal Muscular Atrophy of Type 2 or 3
Assistance Publique - Hôpitaux de Paris 19
Not applicableEnrolling By InvitationNCT07072676
What this trial is testing

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

Who this might be right for
Inclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more
LMU Klinikum 30
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 completedNCT06864767
What this trial is testing

Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA

Who this might be right for
Spinal Muscular Atrophy Type I
Zeynep HOŞBAY 23
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 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
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
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 applicableLooking for participantsNCT06152302
What this trial is testing

Test of Aquatic Mobility of SMA Infants

Who this might be right for
Infantile Spinal Muscular Atrophy
Assistance Publique - Hôpitaux de Paris 15
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 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
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 applicableActive Not RecruitingNCT06191354
What this trial is testing

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

Who this might be right for
Spinal Muscular Atrophy 1
Kun Sun 12
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
Large-scale testing (Phase 3)Ended earlyNCT02193074
What this trial is testing

Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy
Biogen 122
Very early researchStudy completedNCT06576388
What this trial is testing

Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I

Who this might be right for
Type I Spinal Muscular Atrophy
Hangzhou Jiayin Biotech Ltd 2
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
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