Muscular Dystrophy

With the FDA approval, vamorolone (Agamree) becomes the first-in-class dissociative steroid therapy for patients with Duchenne muscular dystrophy. Catalyst Pharmaceuticals plans to launch the treatment in the first quarter of 2024.

In preclinical data, treatment with PGN-EDODM reduced pathogenic nuclear foci by 54% per nuclei and liberated muscleblind like splicing regulator 1, resulting in a greater than 68% correction of downstream transcript missplicing events.

The chief medical officer at ML Bio Solutions, an affiliate of BridgeBio, talked about the significant progress of investigational agent BBP-418 to treat limb-girdle muscular dystrophy type 2I/R9. [WATCH TIME: 3 minutes]

Marketed as Elevidys, the gene therapy is approved for ambulatory pediatric patients aged 4 to 5 with Duchenne based on expression of micro-dystrophin.

At the 2023 MDA conference, the professor of neurology at the University of Minnesota talked about the current understanding and challenges of Limb Girdle muscular dystrophy from a clinical perspective. [WATCH TIME: 5 minutes]