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 »
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 meeting provides an opportunity for SMA scientists and clinicians to share their most recent discoveries and learnings, and is an incubator for collaborations, including cross-disciplinary
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 of risdiplam Real-world evidence generation Check out the full community statement here.
Genentech Releases SMA Community Letter Read More »
