Survey Alert: SMA Family Caregivers
Family members of people with SMA play an active role in managing the daily lives of their loved one – from physical tasks like feeding, bathing, dressing, and more, to mentally-taxing activities like ...
Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of apitegromab in those living with Spinal Muscular Atrophy ...
Cure SMA Discusses FDA-Approved SMA Treatments & Dissects Combination Therapy
Cure SMA's Jacqueline Glascock, Director of Research Programs, recently talked with Managed Healthcare Executive about information on the three FDA-approved SMA treatments and the need for combination therapy. Check out the interview here! Importance ...
Community Update from Novartis Gene Therapies
We are pleased to share a community update on Novartis Gene Therapies' Clinical Trial Program.
Update on Genentech/Roche Initiation of MANATEE Clinical Study
We are pleased to share an update on Genentech's, a member of the Roche Group, initiation of MANATEE, a new global Phase 2/3 clinical study that aims to evaluate the safety and efficacy ...
New Data Presented at World Muscle Society 2021 Virtual Congress
This week, new data was presented at the World Muscle Society 2021 Virtual Congress. Check it out below! Scholar Rock presents two posters featuring additional analyses from the TOPAZ phase 2 ...
Community Statement from Biogen: New Clinical Study Planned to Evaluate the Potential Benefit of an Investigational Higher Dose of Nusinersen in People with SMA Previously Treated with Evrysdi® (risdiplam)
Biogen is committed to improving health outcomes for people impacted by spinal muscular atrophy (SMA) and generating data to assess if there are treatment pathways to address potential unmet needs. Today, we are ...
Cure SMA Convenes Working Group to Discuss Biomarkers for SMA
The advent of three approved SMN-upregulating therapeutics for spinal muscular atrophy has dramatically changed the natural history of the disease. There are, however, many uncertainties regarding timing of treatment initiation, response to treatment, ...
Thank You for Another Fantastic Virtual SMA Conference!
Cure SMA would like to thank everyone who attended the recent 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting. It was an impactful week of workshops, networking, and SMA community engagement. ...
Financial Planning with a Disability
When you first learned that you or your child had SMA, you probably began analyzing your home life, your or your child’s education, and other immediate concerns. In time, you realized that the ...
Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference
Biogen Inc., today announced new research supporting the continued development of an investigational higher dose of SPINRAZA® (nusinersen) and additional data reinforcing the strength of SPINRAZA’s clinical profile in improving the lives of ...
Understanding the Benefits of Aqua Therapy
Each year at the Annual SMA Conference, the session on Aquatic Therapy is among the most popular for attendees of all ages. Jennifer Martyn, PT, is one of the leaders of this educational ...