Discusses HOPE-3 Phase III Trial Results and Regulatory Path for Duchenne Muscular Dystrophy Therapy December 17, ...

As people age, their immune system function declines. T cell populations become smaller and can't react to pathogens as ...

Determining which patients should receive the treatment will still require individual risk-benefit assessments, experts say.

Loss of muscle mass, also called muscle wasting or atrophy, can happen gradually as part of getting older or more suddenly ...

The story so far: The Nobel Prize season for 2025 began with the announcement of the Physiology or Medicine Prize on October 6. The three awardees — U.S.-based researchers, Mary E. Brunkow and Fred ...

The 2025 Nobel Prize in physiology or medicine has been awarded to a trio of scientists – two of them American and one Japanese – for unraveling how the immune system protects us from thousands of ...

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 ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

Findings published in The American Journal of Pathology identify GLUD1 enzyme as a potential therapeutic target for muscle restoration through metabolic reprogramming, addressing clinically unmet need ...

GNT0004 product confirms its clinical efficacy with stabilization of motor functions in patients treated at the effective dose for up to 2 years. Sustained significant reduction in levels of creatine ...