Patient Engagement
Partnership for Spinal Muscular Atrophy (SMA)
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.
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