Front Page 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 […]
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 […]
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 […]
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 […]
Thank You for an Amazing 2023 Annual SMA Conference!
Thank you to everyone who attended the 2023 Annual SMA Conference at Disney’s Yacht & Beach Club in Orlando, Florida! It was an impactful weekend […]
Big Week in Washington for Cure SMA-Supported Legislation
This week, bills to address everyday challenges faced by individuals with spinal muscular atrophy (SMA) took important steps forward in the U.S. Congress. A key […]
Cure SMA’s 2022 State of SMA Report
Cure SMA is pleased to announce the launch of the second annual State of SMA report. The purpose of this report is to share highlights […]
Individuals with SMA Describe New Travel Hiccups and Wheelchair Damage in Their Air Travels to Cure SMA National Conference
A record-number of individuals with spinal muscular atrophy (SMA), a neuromuscular disease, travelled to Orlando to attend Cure SMA’s national conference from June 29 to […]
Luke 18:1 Foundation Donates $500,000 to Cure SMA to Support Groundbreaking SMA Research and Care
Chicago, June 30, 2023 – Cure SMA receives a surprise gift in the amount of $500,181 from the Luke 18:1 Foundation, a 501(c)3 organization founded […]
Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA
• Alcyone’s ThecaFlex DRx™ System is an implantable medical device in development to enable routine subcutaneous administration of therapeutics to the cerebrospinal fluid• First stage […]

