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.
April 10th 2024
The executive vice president and chief research officer of the Muscular Dystrophy Association shared her reaction to the recent FDA approval of givinostat for Duchenne muscular dystrophy.
Post Hoc Analysis Shows Consistent Efficacy With Givinostat for DMD
March 5th 2024A recent post hoc analysis of the phase 3 EPIDYS trial presented at MDA 2024 revealed significant positive outcomes with givinostat, a histone deacetylase inhibitor, among patients with Duchenne muscular dystrophy.
Advancements and Challenges in Genetic Treatments for Neuromuscular Diseases: John W. Day, MD, PhD
February 12th 2024Ahead of the 2024 MDA Conference, the medical advisor and care center director at Muscular Dystrophy Association talked about the track sessions he will be cochairing at the meeting. [WATCH TIME: 5 minutes]
Positive Phase 2 Data Announced for MOMENTUM Study of Duchenne Agent SRP-5051
January 30th 2024Over a 28-week treatment period, the investigational agent demonstrated significantly greater increase in dystrophin expression and exon skipping than previously approved eteplirsen, Sarepta’s first therapy for Duchenne muscular dystrophy.
Dyne Reports Positive Phase 1/2 Data for Duchenne Agent DYNE-251
January 6th 2024After 6 months of treatment, once every 4-week administration of DYNE-251 reached levels of dystrophin expression, exon skipping, and percent dystrophin positive fibers that exceeded levels reported in a previous trial of eteplirsen, considered the standard of care.
Clinical Potential of AOC 1044 in Treating Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping
January 2nd 2024Steve Hughes, MD, chief medical officer at Avidity Biosciences, provided commentary on recently announced positive topline data for AOC 1044, an investigational agent for Duchenne muscular dystrophy.
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]