Discover the forefront of gene therapy at the 4th Gene Therapy for Muscular Disorders Summit (April 16-18, Boston). Following the groundbreaking approval of Sarepta's DMD gene therapy in June 2023, ...

When it comes to muscular diseases, most of us have heard of especially common ones like muscular dystrophy and Lou Gehrig's disease. But one of the rarest muscular disorders is also one of the most ...

I've been blogging for a few years about misophonia, an auditory/neurological disorder that I have advocated for because I and (more importantly) one of my adult children are affected. Recently, at ...

The past twelve months have seen an unprecedented level of excitement, investment, and progression within the gene therapy for muscular disorders field. As the industry endeavours to move gene therapy ...

A team has discovered a possible therapeutic approach to repair injured muscles either from aging or degenerative muscle disorders. For millions of people, losing muscle isn't just about weakness; ...

Congenital muscular dystrophies (CMDs) comprise a heterogeneous group of inherited disorders primarily affecting muscle integrity and function, often with accompanying brain and ocular abnormalities.

U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and ...

Elevated ccf-DNA levels correlate with severe muscle disease in dermatomyositis (DM) The cGAS/STING pathway is key in inflammation, especially in patients with PFA Targeting this pathway may offer new ...