Muscular Dystrophy

After 2 years of treatment, EDG-5506, otherwise known as sevasemten, was well-tolerated, with rapid and sustained decreases in biomarkers of muscle damage.

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.

The executive medical lead at Italfarmaco SpA expressed his confidence in givinostat to mitigate the progression of Duchenne muscular dystrophy, emphasizing its multifactorial mechanism of action. [WATCH TIME: 5 minutes]

The executive medical lead at Italfarmaco SpA talked about data from both animal models and clinical trials supporting the efficacity of givinostat in patients with Duchenne muscular dystrophy at MDA 2024. [WATCH TIME: 5 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Sharon Hesterlee, PhD. [LISTEN TIME: 10 minutes]

The chief executive officer at Elixirgen Therapeutics discussed the company’s mRNA as a promising therapeutic avenue for Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]

The professor of neurology at Vanderbilt University Medical Center discussed advocating for reinstating personalized resources, expanding genetic testing, and improving transitional care in muscular dystrophy. [WATCH TIME: 5 minutes]

The president and chief executive officer of Satellos Bioscience provided an overview of the previously observed preclinical data supporting SAT-3247 as a potential therapy for muscular dystrophies. [WATCH TIME: 4 minutes]

The second-year PhD student in bioinformatics at Boston University who lives with LAMA2 congenital muscular dystrophy talked about the potential impact and challenges of gene therapy in neuromuscular diseases. [WATCH TIME: 5 minutes]

The neuromuscular specialist at the Neurology & Neuromuscular Care Center discussed the pressing need to address the transition of care from pediatric to adult healthcare systems in Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]

A patient advocate within the ALS community discussed the importance of incorporating patients and their voices into clinical meetings and trial design to ultimately meet treatment goals. [WATCH TIME: 4 minutes]

Branded as Duvyzat, givinostat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.

The chief executive officer at Elixirgen Therapeutics provided commentary on the promise of the Bobcat mRNA, and the mechanism of action for mRNA-like therapies to treat Duchenne muscular dystrophy.

The president and chief executive officer of Satellos Bioscience provided insight on a new small molecule therapy, SAT-3247, in development for patients with muscular dystrophies. [WATCH TIME: 3 minutes]

A recent study presented at MDA 2024 highlighted the evolving respiratory patterns in pediatric patients with Duchenne muscular dystrophy, offering crucial insights for effective respiratory management in this patient population.

A recent analysis of data from the US National Registry presented at MDA 2024 revealed gender disparities in pediatric-onset facioscapulohumeral muscular dystrophy, with girls experiencing more severe outcomes.

At MDA 2024, Brooke Eby shared her perspective as a patient with ALS and the ways to incorporate business approaches to improving clinical care and increasing enrollment for drug trials. [WATCH TIME: 5 minutes]

In a recent phase 2 trial analysis of viltolarsen presented at MDA 2024, findings showed improvement in forced vital capacity compared with standard care in patients with Duchenne muscular dystrophy.

A new meta analysis of 3 studies presented at MDA 2024 revealed that ataluren significantly slowed the decline in muscle function for patients with nonsense mutation Duchenne muscular dystrophy.

A 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.

Over a 52-week treatment period, treatment with SRP-9001 resulted in improvements in secondary outcomes of time to rise, micro-dystrophin expression, and 10-meter walk/run.

Patients with Duchenne muscular dystrophy between ages of 6 and 24 months old demonstrated a similar safety profile on eteplirsen than those between 24 and 48 months of age.

In a real-world study, eterplirsen was safe for patients with Duchenne muscular dystrophy, with sustained or improved status in function.

Tadalafil shows potential in addressing microvascular impairment in Duchenne muscular dystrophy, with post-contractile MRI as a screening tool.

In honor of Rare Disease Day, the vice president of public policy and advocacy at the Muscular Dystrophy Association shared his perspective of advocacy and policy progress in the realm of rare neuromuscular diseases. [WATCH TIME: 6 minutes]