Latest News
9th Annual “Hope on the Hill” Congressional Dinner
Just one more week until the 9th Annual “Hope on the Hill” Congressional Dinner in Washington D.C.! Next Tuesday, December 3rd, SMA families and advocates, government officials, and industry leaders […]
Read More ›Cure SMA and Genentech Partner to Provide Travel Support to Adults with SMA for the 2020 Annual SMA Conference
Cure SMA is thrilled to announce that we will, once again, be offering scholarship opportunities to adults with SMA (18 years of age and older) to attend the 2020 Annual […]
Read More ›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, […]
Read More ›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 […]
Read More ›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 […]
Read More ›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). […]
Read More ›