Muscular Dystrophy

Pitolisant demonstrated a dose-dependent improvement in excessive daytime sleepiness and fatigue, with a safety profile that was consistent with previous studies. Additional data to be shared in early 2024.

Givinostat, a HDAC inhibitor, was supported by data from the phase 3 EPIDYS study, a randomized, double-blind, placebo-controlled, multicenter trial.

Despite additional information submitted, the FDA continues to impose a clinical hold on Entrada Therapeutics' ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy.

Ahead of the 2024 MDA Conference, the vice president of public policy and advocacy at the Muscular Dystrophy Association talked about conference advocacy collaboration, access to treatments, and the state of healthcare. [WATCH TIME: 6 minutes]

The director of disability policy at the Muscular Dystrophy Association talked about the future possibility of having an aircraft that accommodates for patients with neuromuscular disorders who use mobility devices. [WATCH TIME: 3 minutes]

The director of disability policy at the Muscular Dystrophy Association discussed his lecture at AANEM 2023 on how clinicians can advocate for their patients to help improve accessibility and health equity. [WATCH TIME: 7 minutes] 

The assistant professor at Virginia Commonwealth University provided comment on her presentation from AANEM 2023, and the need to raise awareness towards nonneuromuscular symptoms of Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]

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]