Latest News
Genentech Roche Releases Community Letter with Clinical Trial Updates
Genentech Roche provided the following community statement with clinical trial updates for SUNFISH (Type 2/3), FIREFISH (Type 1) and JEWELFISH. Dear SMA community, At Genentech (A Member of the Roche […]
Read More ›Cure SMA Releases Schedule for the 2017 Annual SMA Conference
Cure SMA has released the schedule for the 2017 Annual SMA Conference, to be held at Walt Disney World in Orlando, FL, June 29 – July 2. The schedule includes […]
Read More ›Cure SMA Awards $140,000 Grant to Joceyln C̫t̩, PhD, University of Ottawa
Cure SMA has awarded a $140,000 research grant to Joceyln Côté, PhD, at The University of Ottawa, for his project, “Investigating the mechanism by which SMN regulates translation: identification of […]
Read More ›American College of Obstetricians and Gynecologists Recommends Expanded SMA Carrier Screening
The American College of Obstetricians and Gynecologists has released updated guidance on carrier testing for genetic conditions. The updated guidance includes, for the first time, a recommendation that all women […]
Read More ›AveXis Reports Results from Phase 1 Trial of AVXS-101
AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported topline results from the Phase 1 trial of AVXS-101 […]
Read More ›Know Your Resources: How Congressional Caseworkers Can Help You Obtain Insurance Coverage for New Therapies
Your elected officials who represent you in Washington, D.C. have caseworkers in their regional and district offices in your communities. These caseworkers can help you work with and better understand […]
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