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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)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 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 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 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
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 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)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)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 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 applicableLooking for participantsNCT05954455
What this trial is testing

Exploring Bulbar Function, Speech And Communication Development in SMA Type 1

Who this might be right for
SMA1
Institute of Child Health 30
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
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 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 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
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
Guangzhou Jiayin Biotech Ltd 2
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
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 applicableActive Not RecruitingNCT03655223
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 applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
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