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Spinal Muscular Atrophy Clinical Trials

A listing of Spinal Muscular Atrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.


Found (124) clinical trials

Spinraza in Adult Spinal Muscular Atrophy

This is a prospective, longitudinal, multi-center, observational study designed to evaluate the safety, tolerability, and effectiveness of SPINRAZA (nusinersen) in ambulatory and non-ambulatory adult patients with SMA. Subjects with SMA II/III that are 18 years to 60 years of age who are planning to initiate treatment with SPINRAZA (nusinersen) as ...

Phase N/A

Study to Evaluate DNL747 in Subjects With Amyotrophic Lateral Sclerosis

This is a Phase 1b randomized, placebo-controlled, double-blind, crossover study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL747 in subjects with Amyotrophic Lateral Sclerosis (ALS)


Validation Of Tidal/End -Tidal CO2 in ALS

This is a prospective, observational study of patients attending Royal Papworth Hospital's weekly MND clinic. If a patient decides to participate in the study, they will continue to receive all normal care. Researchers will ask permission to review the results of any medical investigations and tests previously undertaken, by looking ...

Phase N/A

Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS

Enrolled patients will be randomized to one of two treatment arms: TUDCA or identical placebo by oral route. The randomization will be performed in a ratio one to one for the two arms. TUDCA will be administered orally at the dose of 1 g twice daily (2 g daily) for ...