We’ve recently released an update to the SMA drug pipeline. This latest version includes: 20 active programs, including three approved therapies. 10 pharmaceutical partners. 12 programs in clinical trials. An ever-increasing breadth of ...
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 ...
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'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 ...
We are pleased to share a community update on Novartis Gene Therapies' Clinical Trial Program.
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 ...
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 ...
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 ...
Every August, the spinal muscular atrophy (SMA) community unites, raises awareness, and shows our pride for SMA Awareness Month. This year, Shaakira Thomas and her family chose to go above and beyond by ...
Novartis announced that the U.S. Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold ...
Data from Genentech, a member of the Roche Group, recently announced that the New England Journal of Medicine (NEJM) has published data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety ...
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, ...
