Neuromuscular

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 on gene therapy for Duchenne muscular dystrophy.

A case series reveals safe use of vamorolone and prednisolone during microdystrophin gene therapy for Duchenne muscular dystrophy in two boys.

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

A recent study reveals the significant economic burden of Angelman syndrome, highlighting rising healthcare costs and ongoing therapeutic developments.

At CNS 2025, the executive director at the Child Neurology Foundation presented on the importance of collaboration among clinicians and advocacy groups to connect families with care support services. [WATCH TIME: 4 minutes]

A post hoc analysis of the phase 3 EPIDYS trial presented at CNS 2025 revealed that givinostat delayed disease progression in 2 distinct age groups of patients with Duchenne muscular dystrophy.

A recent analysis highlights the link between vastus lateralis fat fraction and treatment outcomes in Duchenne muscular dystrophy patients using givinostat.

Yuichi Iwaki, MD, PhD, chief executive officer at MediciNova, spoke on ibudilast’s mechanism of action, its role in the COMBAT-ALS trial, and its potential impact across neurodegenerative disease care.

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]