Latest News
Community Statement from Genentech: FDA Grants Priority Review to Risdiplam for Spinal Muscular Atrophy
Genentech, a member of the Roche Group, today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, […]
SMA Concordance Among Siblings Published in the Journal of Neuromuscular Diseases
Earlier this month, an article was published in the Journal of Neuromuscular Diseases titled, “Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings from the Cure SMA Cohort.” This paper […]
Caring for the Caregiver by Brynne Willis
Many consider the deepest expression of love is being a caregiver. Providing for someone’s needs is incredibly special and rewarding to the one who is giving, as well as the […]
Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy
Scholar Rock today announced preliminary pharmacokinetic (PK) and pharmacodynamic (PD) results from the TOPAZ Phase 2 proof-of-concept trial of SRK-015 for the treatment of patients with spinal muscular atrophy (SMA). […]
Team Cure SMA Raises Over $45,000 in the New York City Marathon
Fifteen Team Cure SMA runners successfully raised over $45,000 for Cure SMA while running in the 2019 TCS New York City Marathon on November 3, 2019. The all-star team included: […]
Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SUNFISH Trial in People With Type 2 or 3 Spinal Muscular Atrophy
Genentech, a member of the Roche Group, today announced positive data from the pivotal Part 2 of the SUNFISH study evaluating risdiplam in people aged 2-25 years with Type 2 […]