(opens in new tab) Scholar Rock  today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for ISEMBYLD™ (apitegromab-mstn) to treat spinal muscular atrophy (SMA) in adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. This approval brings the

From their time at the Cure SMA Annual Conference, to their SMAshing My Limits partnership with Becoming rentABLE, opening up accessible beach houses, treehouses, and yurts for family travel, to adaptive sports and recreation panels, Novartis recaps a busy summer of community connection. Read their latest community update here.

(opens in new tab) NMD Pharma recently shared exciting news with the SMA community: the last participant has completed their part in the SYNAPSE-SMA Phase 2 clinical trial, testing ignaseclant (formerly known as NMD670), an investigational therapy designed to help strengthen skeletal muscles. The trial included 52 adults with SMA Type 3 at sites across