Muscular Dystrophy

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]

PepGen has begun its phase 2 CONNECT1-EDO51 trial, dosing the first patient with PGN-EDO51, an agent for Duchenne muscular dystrophy amendable to an exon 51 skipping.

The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided comments on her session track at the upcoming 2024 MDA conference and some of the recent advancements in ALS research. [WATCH TIME: 4 minutes]

The FDA has accepted Sarepta Therapeutics' efficacy supplement for gene therapy SRP-9001, granting it a priority review for the treatment of Duchenne muscular dystrophy, with a decision expected by June 21, 2024.

The efficacy of vamorolone at a dose of 6 mg/kg/d was maintained over 48 weeks of treatment for all 5 motor outcomes.

The postdoctoral research fellow at Brigham and Women’s Hospital and Harvard Medical School discussed some of the issues with ensuring timely care access to approved therapies for patients with Duchenne muscular dystrophy. [WATCH TIME: 3 minutes]

Despite conditional approval in Europe since 2014, ataluren's authorization has faced rejections and is still investigational in the US.

Despite initial enthusiasm, novel treatments for Duchenne muscular dystrophy face discontinuation challenges and lack long-term clinical efficacy data.