Neuromuscular

The FDA approves the FreedomEDGE infusion platform for rozanolixizumab, enhancing treatment options for generalized myasthenia gravis in clinical settings.

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

Neurology News Network for the week ending January 31, 2025. [WATCH TIME: 4 minutes]

Specifically, the alignment relates to a separate, planned clinical trial referred to as ASPIRE.

New findings from the EMBARK study highlight Elevidys' significant long-term benefits in slowing Duchenne muscular dystrophy progression in young patients.

Intellia’s trial for nex-z in ATTR-CM remains under clinical hold.

A duo of experts from CureDuchenne discussed how the inclusion of Duchenne muscular dystrophy on the Recommended Uniform Screening Panel can enable earlier diagnosis and inform decision-making in a rapidly evolving therapeutic landscape. [WATCH TIME: 7 minutes]

Trial data showed that treatment with lamotrigine reduced myotonia severity in adults with genetically confirmed non-dystrophic myotonias, with sustained benefit and an acceptable safety profile.

The associate chief medical officer at Michigan State University highlighted advances in neuromuscular disease, emerging therapeutic strategies, and the growing role of patient-reported outcomes at AANEM 2025. [WATCH TIME: 2 minutes]

The FDA raised no objections to Lexicon Pharmaceuticals’ pilavapadin for a phase 3 program, which will include 2-arm registrational studies, in patients with diabetic peripheral neuropathic pain.

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

The consultant neurologist at the National Neuroscience Institute discussed how artificial intelligence, biomarker development, and drug repurposing can help improve patient care in ALS. [WATCH TIME: 4 minutes]

Benitec Biopharma reveals promising results for BB-301, a gene therapy showing significant improvements in swallowing function for OPMD patients.

Jerel Banks, MD, PhD, chief executive officer of Benitec Biopharma, reviews early phase 1b/2a clinical signals, mechanism, and next steps for BB-301 in oculopharyngeal muscular dystrophy.

Neurology News Network for the week ending January 24, 2025. [WATCH TIME: 4 minutes]

AskBio noted that AB-1009 also recently received fast track and orphan drug designations from the FDA.

The FDA requested the full data from the phase 3 HOPE-3 study as part of its review of the biologics license application for deramiocel, which Capricor plans to submit in February 2026.

Stephanie Fradette, PharmD, head of neuromuscular development at Biogen, gave immediate reaction to the EC approval of high-dose nusinersen and its implications for evolving SMA treatment strategies.

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

Neurology News Network for the week ending January 17, 2025. [WATCH TIME: 4 minutes]

The therapy also received orphan drug designation from the FDA.

Paul Melmeyer, MPP, executive vice president, public policy and advocacy, Muscular Dystrophy Association, explains how adding Duchenne muscular dystrophy to the Recommended Unified Screening Panel reshapes early diagnosis, treatment timing, and family counseling.

Published phase 3 data in JAMA Neurology highlighted tofersen's potential to slow ALS progression and improve survival, marking a breakthrough in SOD1-ALS treatment.

In the middle of 2026, the company is planning to submit a BLA for RGX-202 via an accelerated approval pathway.

The European Commission approved Biogen’s high-dose nusinersen regimen for 5q SMA, supported by DEVOTE trial data showing significantly improved motor function with a safety profile consistent with the standard-dose regimen.