Neuromuscular

The phase 3 FORTIFY trial of BBP-418 met its primary and key secondary interim analysis end points and demonstrated a safety profile consistent with prior studies in patients with LGMD2I/R9.

The vice president and Asset Head of Rare and Ultra-Rare Diseases at UCB provided clinical perspectives on the FDA approval of Kygevvi, the first marketed treatment specific for patients with thymidine kinase 2 deficiency (Tk2d).

A genetic counselor-only clinic enhances access to genetic testing in pediatric neurology, proving efficient and effective for timely precision diagnostics.

A study reveals the intricate decision-making process caregivers face when considering gene therapy for Duchenne muscular dystrophy, emphasizing shared input and personal experiences.

Although their knowledge, experience, and access to genetic counselors varied, most licensed OB/GYNs across Ohio reported routinely offering carrier screening for spinal muscular atrophy.

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

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 7, 2025.

A new study presented at NSGC 2025 showed that patients living with amyotrophic lateral sclerosis considering medical aid in dying often experienced emotional and logistical challenges with the process.

A recent study highlights the link between genetic neurodevelopmental disorders and increased gastrointestinal issues in pediatric patients, emphasizing the need for targeted care.

Spingogenix presents promising data on SPG302, a daily tablet showing cognitive improvements in ALS patients with excellent safety profiles.

Feedback from the FDA allows CervoMed to move forward with the proposed primary and secondary end point of the phase 3 trial assessing neflamapimod in dementia with Lewy bodies.

FDA issues a complete response letter for Biohaven's troriluzole, a potential treatment for spinocerebellar ataxia, citing data concerns.

The associate professor of physical medicine and rehabilitation at Virginia Commonwealth University discussed his passion for educating others about electrodiagnosis and ultrasound in neuromuscular disorders.

A phase 1/2 trial explores YTB323, a CAR T cell therapy, for treating generalized myasthenia gravis, aiming to enhance patient outcomes and safety.

UCB's Kygevvi becomes the first FDA-approved treatment for thymidine kinase 2 deficiency, offering hope to patients with this rare neuromuscular disorder.

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

The head of Rare Diseases US at UCB talked about the company’s latest data on rozanolixizumab and zilucoplan in patients living with generalized myasthenia gravis. [WATCH TIME: 3 minutes]

The chief medical officer at Mandos Health discussed new CNS 2025 data on adrabetadex in infantile-onset Niemann-Pick type C and the growing evidence of its potential disease-modifying impact.

Neurology News Network for the week ending November 1, 2025. [WATCH TIME: 3 minutes]

A groundbreaking trial compares nipocalimab and efgartigimod for treating generalized myasthenia gravis, promising new insights for patient care.

A perspective model aimed noted that introducing efgartigimod alfa for patients with chronic inflammatory demyelinating polyneuropathy incurs more expected costs.

Patients with generalized myasthenia gravis reported a positive experience with rozanolixizumab self-administration, with a preference for the manual push method.

An open-label extension trial testing the efficacy of nipocalimab in patients transitioning from placebo revealed continued improvement in MG-Activities of Daily Living scores over a long-term period.

Data showed that efgartigimod led to greater improvements in disease activity compared with placebo in idiopathic inflammatory myopathy, and improvements in seronegative myasthenia gravis.

An analysis of the first 110 patients screened for the ARISE study showed that an independent adjudication committee confirmed the diagnosis of CIDP in nearly 3-quarters of cases.