The NeurologyLive® Muscular Dystrophy Disease Spotlight page offers specific coverage on the latest expert conversations and clinical trial data associated with the treatment and management of patients with muscular dystrophies.
September 6th 2024
Treatment with DYNE-251 revealed dose dependent exon skipping and dystrophin expression as well as improvement in multiple functional end points in both cohorts of the phase 1/2 DELIVER trial.
How Clinicians can Advocate for Patients Beyond the Clinic: Michael Lewis, MA
November 7th 2023The 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]
Addressing the Invisible Symptoms of Duchenne Muscular Dystrophy: Mathula Thangarajh, MD, PhD
November 6th 2023The 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]
FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy
October 26th 2023With 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.
FDA Lifts Clinical Hold on FREEDOM-DM1 Study of Antisense Agent PGN-EDODM1
October 14th 2023In 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.
Overcoming the Challenges of Ultra Rare Diseases, Limb Girdle Muscular Dystrophy: Peter Kang, MD
March 30th 2023At 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]