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Results for “Type 2 Spinal Muscular Atrophy”

59 trials

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Showing 20 of 59 results

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 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
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
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 RecruitingNCT03709784
What this trial is testing

Spinraza in Adult Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular AtrophySpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type 3
Washington University School of Medicine 148
Testing effectiveness (Phase 2)Active Not RecruitingNCT05794139
What this trial is testing

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy
NMD Pharma A/S 54
Testing effectiveness (Phase 2)Looking for participantsNCT07070999
What this trial is testing

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Who this might be right for
Spinal Muscular Atrophy Type I
Gemma Biotherapeutics 22
Not applicableUnknownNCT05272969
What this trial is testing

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more
LMU Klinikum 95
Testing effectiveness (Phase 2)Looking for participantsNCT07047144
What this trial is testing

Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular AtrophySMASpinal Muscular Atrophy Type 2+3 more
Scholar Rock, Inc. 52
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 applicableUnknownNCT03300869
What this trial is testing

Natural History of Types 2 and 3 SMA in Taiwan

Who this might be right for
Spinal Muscular Atrophy
Kaohsiung Medical University Chung-Ho Memorial Hospital 300
Not applicableUnknownNCT05839145
What this trial is testing

Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study

Who this might be right for
Muscular Atrophy, Spinal, Type IIMuscular Atrophy, Spinal, Type III
Institut de Myologie, France 60
Testing effectiveness (Phase 2)Study completedNCT03921528
What this trial is testing

An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+6 more
Scholar Rock, Inc. 58
Not applicableStudy completedNCT01033331
What this trial is testing

Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid

Who this might be right for
Spinal Muscular Atrophy
University of Sao Paulo General Hospital 22
Not applicableEnrolling By InvitationNCT06772402
What this trial is testing

GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy Type 2
Genecombio Ltd. 6
Testing effectiveness (Phase 2)Active Not RecruitingNCT05901987
What this trial is testing

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

Who this might be right for
SMA II
GeneCradle Inc 33
Testing effectiveness (Phase 2)Study completedNCT00533221
What this trial is testing

Pilot Study of Growth Hormon to Treat SMA Typ II and III

Who this might be right for
Muscular Atrophy, Spinal
University Hospital Freiburg 20
Not applicableActive Not RecruitingNCT04159987
What this trial is testing

Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA®

Who this might be right for
Spinal Muscular Atrophy
Centre Hospitalier Universitaire de Nice 20
Not applicableEnrolling By InvitationNCT06605612
What this trial is testing

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyLimb-girdle and Facioscapulohumeral Muscular Dystrophies+9 more
LMU Klinikum 108
Testing effectiveness (Phase 2)Study completedNCT00227266
What this trial is testing

Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy
University of Utah 94
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