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 ...

A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been demonstrated by an analysis of data ...

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 ...

DelveInsight’s “Spinal Muscular Atrophy Pipeline Insight 2025” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in the Spinal Muscular Atrophy pipeline landscape. It ...

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 ...

Novartis’ ITVISMA, approved a month ago by the US FDA, used at Sheikh Khalifa Medical City under the supervision of the ...

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented positive results from the primary analysis of the ongoing RAINBOWFISH ...

New York, Feb. 12, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today celebrates the decision by the US Food and Drug Administration (FDA) to grant approval of a risdiplam ...