Neuromuscular

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.

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.

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.

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.

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.

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

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

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.

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.