Neuromuscular

In the MOXIe open-label extension study presented at MDS 2025, omaveloxolone treatment was associated with no significant disease progression in bulbar function or upper limb coordination.

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

Nipocalimab receives positive EMA recommendation, potentially transforming treatment for generalized myasthenia gravis with significant clinical trial results.

The president of the LGMD Foundation discussed raising awareness, strengthening advocacy, and the lived experience of navigating limb-girdle muscular dystrophy.

The founder of the LGMD Foundation provided context on the significance behind LGMD Awareness Day, an initiative dedicated toward uplifting the limb-girdle muscular dystrophy community. [WATCH TIME: 6 minutes]

Melissa Spencer, PhD, discussed the rare and complex landscape of limb girdle muscular dystrophy, highlighting research challenges, safety considerations in gene therapy, and the need for natural history studies and biomarkers.

Kelly Brazzo, founder and chief executive officer of the CureLGMD2i Foundation, provided commentary on LGMD Awareness Day, focusing on subtype-specific advocacy, genetic testing, and platform development leading to approved therapies.

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

Neurology News Network. for the week ending September 27, 2025. [WATCH TIME: 4 minutes]

Biogen plans to resubmit its application for a higher dose of nusinersen after receiving FDA feedback, aiming to enhance SMA treatment efficacy.

Ionis reveals promising trial results for zilganersen, a potential first treatment for Alexander disease, aiming to transform patient care.

The psychiatrist and founder of the Stuttering Treatment and Research Society discussed the historical persistence of stuttering, treatment gaps, and the nuances of managing psychiatric comorbidities. [WATCH TIME: 3 minutes]

Scholar Rock navigates FDA hurdles for apitegromab, a promising treatment for spinal muscular atrophy, aiming for resubmission after addressing manufacturing concerns.

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

The founder of the Stuttering Treatment and Research Society (STARS) detailed current therapeutic strategies for stuttering, the role of dopamine pathways, and the pressing need for FDA-approved options. [WATCH TIME: 4 minutes]

The vice chair of neurology at West Virginia University outlined key challenges in academic neurology and reflected on the evolving responsibilities of vice chairs in supporting departmental growth and leadership. [WATCH TIME: 3 minutes]

Avidity's del-zota shows promising results in reversing disease progression in Duchenne muscular dystrophy, paving the way for potential FDA approval.

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New guidelines enhance gene therapy delivery for Duchenne muscular dystrophy, emphasizing multidisciplinary care and safety monitoring for optimal patient outcomes.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 12, 2025.

Saol Therapeutics faces challenges after FDA's complete response letter for SL1009, a potential treatment for rare mitochondrial disorder PDCD.

The chief medical adviser at the Muscular Dystrophy Association spoke about the specific safety considerations clinicians should take when prescribing and administering gene therapy for patients with Duchenne muscular dystrophy. [WATCH TIME: 3 minutes]

Catch up on any of the neurology news headlines you may have missed in August 2025, compiled into 1 place by the NeurologyLive® team.

New trial data reveal claseprubart shows significant efficacy in treating generalized myasthenia gravis, paving the way for potential FDA approval.

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