For Patients

Partnership for Spinal Muscular Atrophy (SMA)

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CureSMA

CureSMA funds and directs research paving the way for treatment and care for those affected by spinal muscular atrophy, while also promoting a supportive community. Through collaborative endeavors between researchers, practitioners, families, and the patients themselves, CureSMA takes a comprehensive approach to their strategy of developing a treatment.

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CLINICAL TRIAL

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy (DEVOTE)

PI: Basil Darras, MD  |  Clinical Drug Trial — NCT04089566 >
Patient Advocacy Group Partners: Cure SMA, Muscular Dystrophy Association

CLINICAL TRIAL

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)

PI: Basil Darras, MD  |  Clinical Drug Trial — NCT04851873 >
Patient Advocacy Group Partners: Cure SMA, Muscular Dystrophy Association

CLINICAL TRIAL

Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy (FIREFISH)

PI: Basil Darras, MD  |  Clinical Drug Trial — NCT02913482 >
Patient Advocacy Group Partners: Cure SMA, Muscular Dystrophy Association

CLINICAL TRIAL

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy (Jewelfish)

PI: Basil Darras, MD  |  Clinical Drug Trial — NCT03032172 >
Patient Advocacy Group Partners: Cure SMA, Muscular Dystrophy Association

CLINICAL TRIAL

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulatory Children With Spinal Muscular Atrophy

PI: Basil Darras, MD  |  Clinical Drug Trial — NCT05115110 >
Patient Advocacy Group Partners: Cure SMA, Muscular Dystrophy Association

CLINICAL TRIAL

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

PI: Basil Darras, MD  |  Clinical Drug Trial — NCT03921528 >
Patient Advocacy Group Partners: Cure SMA, Muscular Dystrophy Association