Front Page News
Check Out the Cure SMA Booklet on “Scientific Considerations for Drug Combinations”
There are now two treatments approved by the U.S. Food and Drug Administration (FDA) that target the underlying genetics of spinal muscular atrophy (SMA) and […]
Read More ›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, […]
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) […]
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 […]
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 […]
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 […]
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 […]
Read More ›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, […]
Read More ›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 […]
Read More ›A Tiny Warrior: Winnie Stanley’s Story
This guest post is written by April Leiffer Henry If you ask 4-year-old Winnie Stanley what she wants to be when she grows up, she’ll […]
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