Researchers have identified a protein that’s critical to the development of skeletal muscle mass and strength and the tissue’s ability to metabolize glucose. They say their findings may lead to ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study ...

Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease. This is a ...

Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important ...

Ancient bacteria that have evolved to become integral to our cells – converting nutrients from food into energy – may also contribute to neurologic disorders, such as spinal muscular atrophy (SMA) and ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...

Scholar Rock's biologics license application for apitegromab was accepted by the Food and Drug Administration. The late-stage biopharmaceutical company said the regulator will review the application ...

NHS pilot would cure babies with spinal muscular atrophy (SMA) in some parts of the UK but not others - but Wes Streeting as ‘asked his team’ if all babies could be saved ...