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 safety of Evrysdi® (risdiplam) in babies with pre-symptomatic SMA, aged from birth to 6 weeks. These data were presented at the 28th World Muscle Society (WMS) Congress. Read more about […]
Genentech Shares 1-Year Primary Analysis from RAINBOWFISH Read More »
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 year, and we look forward to seeing some of you again at the Cure SMA Chapter and Leadership Meeting. In this community update we are pleased to share a Plain
Novartis Gene Therapies SMA Community Update Read More »
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 adults and family members with SMA who are in Washington, D.C. to help advocate for the SMA community. On Thursday, our group of SMA community leaders from across the country
Cure SMA on Capitol Hill – A Message from Allie Williams Read More »
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 community and the future we’re building together. We are grateful for those living with SMA and loved ones who share their experiences with others and help inspire our work, the
SMA Highlights and Updates from Biogen Read More »
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 with SMA. We recently published a new research article, “Identifying Biomarkers of Spinal Muscular Atrophy for Further Development.” This article appeared in the July 12, 2023 issue of the peer-reviewed
Identifying Biomarkers of Spinal Muscular Atrophy for Further Development Read More »
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 been dosed in their Phase II clinical trial of the CIC-1 inhibitor NMD670. To read the full press release, visit this link. MD670 is a first-in-class small molecule inhibitor of
NMD Pharma Initiates Phase II Trial for SMA Read More »
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 Types 2 and 3 spinal muscular atrophy (SMA) who are receiving SMN therapy (either nusinersen or risdiplam). Scholar Rock also announced that it plans to
Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial Read More »
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 Taldefgrobep and the Phase 3 Trial Taldefgrobep is an investigational, muscle-targeted recombinant protein with the potential to enhance muscle mass and strength in people living
Biohaven Completes Enrollment in Pivotal Phase 3 Study Read More »
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 is designed to focus on a topic that is currently of great interest to both researchers and clinicians. This year’s Special Session, “The State of
The State of SMA – Toward Improved Therapeutics and Care Read More »
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 self-acceptance, she is confident that living with SMA helps her relate to students on a deeper level. “Just because there are limits that a diagnosis
Community Spotlight: Sarah Manuel Read More »
