Neuromuscular Disease News

NeuroSense Therapeutics Ltd. has reported positive results from the 12-month data analysis of a phase 2b study evaluating a novel extended-release oral formulation combination therapy in patients with amyotrophic lateral sclerosis.

Certain physical exercises may prevent nerve damage caused by chemotherapy among individuals being treated for cancer, results of a randomized study published in JAMA Internal Medicine showed.

Avidity Biosciences Inc. has announced positive data from the phase 1/2 FORTITUDE clinical trial of a proprietary monoclonal antibody that binds to the transferrin receptor 1 conjugated with a siRNA targeting DUX4 mRNA.

DENVER — The use of several common antibiotics led to exacerbation of myasthenia gravis in a small cohort of patients, Sanem P. Uysal, MD, said in this Healio video from the American Academy of Neurology annual meeting.

Wave Life Sciences has announced positive results from its phase 1b/2a clinical trial of WVE-003, a potential disease-modifying therapeutic for Huntington’s disease.

The FDA has announced approval of a label expansion for Elevidys to include patients with Duchenne muscular dystrophy aged 4 years and older with a confirmed mutation in the DMD gene.

The FDA has approved Vyvgart Hytrulo in a once-weekly, 30- to 90-second subcutaneous injection to treat adults with chronic inflammatory demyelinating polyneuropathy, according to the manufacturer.

The FDA has approved an abbreviated new drug application for a generic version of Emflaza to treat Duchenne muscular dystrophy, according to the manufacturer.

Newly released data from the open-label extension of the FIREFISH study confirmed sustained safety and efficacy of Evrysdi in children with type 1 spinal muscular atrophy over 5 years.

Prothena Corp. recently announced it would receive $80 million from Bristol Myers Squibb for an exclusive global license to an investigational therapeutic developed to address protein dysregulation.