Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 16, 2024.

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.

Experts from different subspecialties in neurology shared their clinical perspectives for patients on promising therapeutics and expansion of care interventions for 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.

Omaveloxolone is an oral, once-daily medication indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older in both the United States and the European Union.

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]

Ahead 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]

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is neuromuscular disorders, and specifically, Duchenne muscular dystrophy.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 9, 2024.

The postdoctoral research fellow at Brigham and Women’s Hospital and Harvard Medical School provided answers to a recently published study highlighting the burden of care and infrequent use of novel exon-skipping drugs for Duchenne in clinical settings. [WATCH TIME: 3 minutes]

The chief executive officer of ClearPoint Neuro provided clarity on how the recently approved SmartFrame system can enhance the efficiency of operating room procedures, allowing for flexible workflows and image-based corrections.

The promising results of nipocalimab in pivotal studies suggest a potential breakthrough in treating autoimmune diseases like generalized myasthenia gravis and Sjögren’s.

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is stroke.

Neurology News Network for the week ending February 3, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 2, 2024.

The adjunct professor of human genetics at Emory University School of Medicine discussed previous research on Rett syndrome that initially provided crucial insight on the neuromuscular condition in adult populations. [WATCH TIME: 2 minutes]

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

VCA-894A, a novel antisense oligonucleotide, exhibits a mechanism of action that specifically targets a cryptic splice site variant within immunoglobulin mu-binding protein 2.

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

Over 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.

Take a look at some of the most-anticipated FDA pending approvals expected in 2024 that researchers and clinicians in neurology should keep an eye out on.

Takeda's FDA-approved Gammagard Liquid can be used as induction therapy, which includes an induction dose followed by maintenance doses, for adults with chronic inflammatory demyelinating polyneuropathy.

Here's some of what is coming soon to NeurologyLive® this week.