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Results for “SMA - Spinal Muscular Atrophy”

343 trials

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Showing 20 of 343 results

Not applicableWithdrawnNCT01663584
What this trial is testing

Multi-disease Carrier Screening Test Validation

Who this might be right for
Spinal Muscular Atrophy (SMA)Carrier ScreeningGenetic Testing
Natera, Inc.
Early research (Phase 1)Active Not RecruitingNCT05575011
What this trial is testing

Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec

Who this might be right for
Healthy VolunteerMuscular Atrophy, Spinal
Biogen 62
Testing effectiveness (Phase 2)Study completedNCT02628743
What this trial is testing

Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)

Who this might be right for
Muscular Atrophy, Spinal
Hoffmann-La Roche 131
Not applicableStudy completedNCT01059240
What this trial is testing

SMN Copy Number Distribution in Mali, West Africa

Who this might be right for
Spinal Muscular Atrophy
National Institute of Neurological Disorders and Stroke (NINDS) 701
Not applicableLooking for participantsNCT03555578
What this trial is testing

Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)"

Who this might be right for
Spinal and Bulbar Muscular Atrophy
Takeda 1,890
Not applicableLooking for participantsNCT07223489
What this trial is testing

Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System

Who this might be right for
Spinal Muscular Atrophy (SMA)
Medstar Health Research Institute 200
Not applicableUnknownNCT00154960
What this trial is testing

Establishing Novel Detection Techniques for Various Genetic-Related Diseases by Applying DHPLC Platform.

Who this might be right for
Spinal Muscular AtrophyNeonatal HyperbilirubinemiaColon Cancer
National Taiwan University Hospital 1,000
Not applicableStudy completedNCT03554343
What this trial is testing

Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium

Who this might be right for
Spinal Muscular Atrophy
Centre Hospitalier Régional de la Citadelle 136,339
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)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 applicableLooking for participantsNCT04875416
What this trial is testing

Phenotype, Genotype and Biomarkers 2

Who this might be right for
Amyotrophic Lateral SclerosisHereditary Spastic ParaplegiaPrimary Lateral Sclerosis+2 more
University of Miami 300
Early research (Phase 1)Temporarily pausedNCT01337518
What this trial is testing

A Phase 1a/1b Study to Evaluate the Safety of EZN-4176, in Adult Patients With Castration-Resistant Prostate Cancer

Who this might be right for
Prostatic Neoplasm
Enzon Pharmaceuticals, Inc. 150
Not applicableActive Not RecruitingNCT04690998
What this trial is testing

Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients

Who this might be right for
Spinal Muscular Atrophy
Assistance Publique Hopitaux De Marseille 104
Not applicableLooking for participantsNCT06315673
What this trial is testing

Digital Assessment of Speech and Fine Motor Control in ALS

Who this might be right for
Amyotrophic Lateral SclerosisPrimary Lateral SclerosisProgressive Muscular Atrophy
Milton S. Hershey Medical Center 50
Post-approval studies (Phase 4)Active Not RecruitingNCT05232929
What this trial is testing

Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Who this might be right for
Spinal Muscular Atrophy
Genentech, Inc. 404
Not applicableStudy completedNCT01754441
What this trial is testing

Mechanisms of Cell Death in Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy
Nemours Children's Clinic 25
Testing effectiveness (Phase 2)Study completedNCT03437941
What this trial is testing

Study to Evaluate CORT125281 in Combination With Enzalutamide in Patients With mCRPC

Who this might be right for
Metastatic Castration-Resistant Prostate Cancer
Corcept Therapeutics 39
Testing effectiveness (Phase 2)Ended earlyNCT04926181
What this trial is testing

Apalutamide Plus Cetrelimab in Patients With Treatment-Emergent Small Cell Neuroendocrine Prostate Cancer

Who this might be right for
Small Cell Neuroendocrine CarcinomaProstate CancerSmall Cell Carcinoma
Rahul Aggarwal 2
Not applicableUnknownNCT04262570
What this trial is testing

Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)

Who this might be right for
Muscular DiseasesSpinal Muscular Atrophy
University of Erlangen-Nürnberg Medical School 10
Not applicableLooking for participantsNCT00874783
What this trial is testing

Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases

Who this might be right for
Neurodegenerative Disorders
Hadassah Medical Organization 120
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