Latest News
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 apitegromab in nonambulatory patients with […]
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 official press release here. About […]
Read More ›The State of SMA – Toward Improved Therapeutics and Care
Each year, Cure SMA collaborates with our Scientific Advisory Board to plan a Special Session that will be held during the Annual Research & Clinical Care Meeting. This Special Session […]
Read More ›Community Spotlight: Sarah Manuel
In Sarah Manuel’s career as a school psychologist, she mentors elementary, middle and high school students as they navigate everyday challenges in life and education. Though it took time and […]
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 Care Meeting. The annual […]
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 gene-therapy Long-term efficacy and safety […]
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