Introduction Rare diseases (RDs) affect millions of people worldwide and present major challenges for research due to small ...

Engineered extracellular vesicles delivered full-length Duchenne muscular dystrophy messenger RNA to skeletal muscle in preclinical models, restoring dystrophin production without serious toxicity A ...

Multiple sclerosis (MS) affects the protective myelin sheath around nerve fibers in the brain and spinal cord, while muscular dystrophy (MD) is a group of genetic conditions that damage and destroy ...

Duchenne muscular dystrophy (DMD) is caused by a mutation in the DMD gene that prevents dystrophin production, leading to progressive muscle weakness and early mobility challenges. The 2018 DMD ...

This study reveals abnormal iron accumulation in the muscles of patients with Facioscapulohumeral Muscular Dystrophy (FSHD) and in mice expressing muscle-specific DUX4 (DUX4-Tg mice). Contrary to ...

The peripheral nervous system (PNS) is involved in nociception and gait. The contribution of PNS-resident immune cells to these functions is not fully understood. We identified group 2 innate lymphoid ...

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.

For patients with nonambulatory type 2 or type 3 spinal muscular atrophy, apitegromab, a monoclonal antibody that inhibits myostatin activation, improves motor function versus placebo, according to a ...

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...

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 ...