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 […]
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 […]
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 […]
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 […]
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 […]
Key Congressional Committee Urges NIH to Address Unmet Needs of Individuals with SMA
On July 27th, the U.S. Senate Appropriations Committee supported a Cure SMA-requested provision asking the National Institutes of Health (NIH) to address the unmet needs of individuals with spinal muscular […]