Neuromuscular

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.

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.

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.

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.

AHN's neuro-oncology program aims to revolutionize brain and spine tumor care with innovative therapies, personalized treatment plans, and advanced research initiatives.

Subgroup data from a phase 2 CIDP trial showed riliprubart maintained consistent efficacy and safety across patient demographics, disease characteristics, and prior treatment exposures.

Findings suggest a potential correlation between motor function improvement and decreased autophagy-related protein levels in patients with spinal muscular atrophy treated with nusinersen.

A newly presented study showed that patients with sarcopenia before starting chemotherapy had a greater likelihood of developing moderate to severe peripheral neurotoxicity.

Eplontersen showed promise in halting disability progression and improving gait speed in patients with hereditary amyloid polyneuropathy, according to recent study findings.

A recently presented biobank data analysis showed associations between reduced transthyretin and higher incidence of conditions such as Alzheimer disease, cardiovascular disease, and rheumatoid arthritis.

Among patients with severe Guillain-Barré syndrome, imlifidase plus IVIg resulted in improved mobility, strength, and disability scores, with sustained benefit over a 6-month time period.

A recently presented matching-adjusted indirect comparison assessed outcomes of immune globulin subcutaneous (Human)-ifas, 10% solution versus subcutaneous efgartigimod in patients with CIDP.

Carba1 showed promise in preclinical models as a dual-action agent, protecting against chemotherapy-induced neuropathy while preserving anticancer efficacy and safety.

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

Presented at the 2025 PNS Annual Meeting, phase 2 ARDA data showed empasiprubart reduced IVIg retreatment risk and improved grip strength in patients with MMN.