Front Page News
Genentech Shares 1-Year Primary Analysis from RAINBOWFISH
Last week, Genentech, a member of the Roche Group, presented positive results from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and […]
Read More ›Novartis Gene Therapies SMA Community Update
Dear SMA Community, It was a pleasure to be able to join so many of you at the Cure SMA Annual Conference in Orlando this […]
Read More ›Cure SMA on Capitol Hill – A Message from Allie Williams
Hey, it’s Allie here from Oklahoma! I am privileged to serve on the Cure SMA Adult Advisory Council and am part of a group of […]
Read More ›SMA Highlights and Updates from Biogen
Dear SMA Community, As we reflect on SMA Awareness Month, we take this opportunity to celebrate all those who are a part of the SMA […]
Read More ›Identifying Biomarkers of Spinal Muscular Atrophy for Further Development
An important part of Cure SMA’s core mission is to carry out and support research that will improve the quality of life for all people […]
Read More ›NMD Pharma Initiates Phase II Trial for SMA
NMD Pharma, a clinical-stage biotech company leading the development of novel first-in-class therapies for severe neuromuscular disorders, recently announced that the first SMA patient has […]
Read More ›Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial
Today, Scholar Rock announced that enrollment in their Phase 3 SAPPHIRE trial is now complete. The Phase 3 trial will evaluate the safety and efficacy of […]
Read More ›Biohaven Completes Enrollment in Pivotal Phase 3 Study
Recently, Biohaven released a statement on the completed enrollment of their pivotal Phase 3 Study of Taldefgrobep Alfa in people with SMA. Check out Biohaven’s […]
Read More ›Clinical Research and Drug Development Session Highlights from 2023 SMA Research & Clinical Care Meeting
At the end of June, SMA researchers and clinicians from around the world met in Orlando, Florida, for the 2023 Annual SMA Research & Clinical […]
Read More ›Genentech Releases SMA Community Letter
Genentech recently issued an SMA community statement highlighting updates from the 2023 SMA Research & Clinical Care Meeting. These updates included: Investigating risdiplam treatment post […]
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