Neuromuscular

Neurology News Network. for the week ending June 7, 2025. [WATCH TIME: 4 minutes]

New data reveals RGX-202 gene therapy shows significant improvements in Duchenne muscular dystrophy patients, enhancing disease management and safety.

Phil Lambert, PhD, chief scientific officer at Satellos Bioscience, discusses the rationale and early clinical progress of SAT-3247, a novel regenerative therapy targeting muscle stem cells in Duchenne muscular dystrophy.

AJ201 shows promising safety and efficacy in treating spinal and bulbar muscular atrophy, marking a potential breakthrough for patients.

Josh Bryson, PhD, head of Medical Affairs at Argenx, discussed the safety profile of efgartigimod in light of recent FAERS-based infection data and offered clinical context for patient care.

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

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

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 30, 2025.

AHN Saint Vincent Hospital offers advanced neurological care, reducing wait times and providing local access to specialized treatments for various conditions.

Jeff Smith, PhD, chief executive officer at Precision Biosciences, explained the clinical potential and gene-editing mechanism of PBGENE-DMD, a one-time therapy targeting dystrophin mutations in Duchenne muscular dystrophy.

The vice president and franchise lead of Autoimmune at Hansa Biopharma shed insights on imlifidase’s targeted mechanism and promising phase 2 data in treating Guillain-Barré syndrome.

The vice president and franchise lead of Autoimmune at Hansa Biopharma provided clinical insights on latest phase 2 safety and efficacy data on imlifidase, an IgG-cleaving antibody, in Guillain-Barré syndrome. [WATCH TIME: 3 minutes]

Nuvig's NVG-2089 shows promise in CIDP treatment, offering a safer, more efficient alternative to IVIg with a patient-friendly trial design.

BioVie initiates a phase 2 trial for bezisterim, targeting neurological symptoms of Long COVID, aiming to improve patient quality of life.

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

Investigators explore a phase 4 trial transitioning CIDP patients from IVIg to efgartigimod, aiming to enhance treatment safety and efficacy.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 23, 2025.

A recent study suggests rituximab may benefit CIDP patients, showing improvement in neurofilament levels and clinical outcomes after treatment.

Researchers reported that both EN001 and insulin improved muscle strength and nerve function in models of Charcot-Marie-Tooth disease type 1A, with combined treatment showing enhanced effects.

Newly presented findings from the NEURO-TTRansform study identified thresholds for meaningful improvement in symptoms and disability, which were exceeded by eplontersen treatment.

New findings confirm vutrisiran's long-term safety and efficacy in treating hereditary transthyretin amyloidosis, enhancing patient quality of life.

The vice president and franchise lead of Autoimmune at Hansa Biopharma discussed the rapid, antibody-cleaving mechanism of imlifidase and its potential to address unmet needs in Guillain-Barré syndrome. [WATCH TIME: 3 minutes]

Gene therapy arsa-cel significantly improves nerve function in children with late-infantile metachromatic leukodystrophy, outperforming traditional treatments.

A recent matched cohort analysis revealed that patients with CIDP treated with immunoglobulin had lower rates of assistive device deterioration and opioid use compared with those untreated.

Matched analysis from the IGOS dataset showed ANX005-treated patients had greater improvements in GBS disability scores over time compared with those receiving IVIg or plasma exchange.