Neuromuscular Disease News

A clinical-stage biopharma firm announced it has been granted a U.S. patent expanding coverage of a novel oral therapy to treat various conditions associated with loss-of-function mutations of the gene encoding methyl-CpG binding protein.

A clinical-stage biopharmaceutical company has reported new positive data from a 12-month long-term open label extension of the CNM-Au8 treatment arm in the HEALEY ALS Platform Trial.

Healio Neurology looks back at the most-viewed stories throughout the year. Highlights include the FDA’s approval of Leqembi to treat Alzheimer’s disease and the regulatory body’s clearing of a blood test for concussions.

A pharmaceutical company and a biotechnology firm have announced a development and license agreement for an investigational proprietary biologic combination therapy to treat amyotrophic lateral sclerosis.

A clinical-stage RNA medicines firm has announced the initiation of dosing in a phase 2 clinical trial of its investigational therapy for boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping.

A committee of the European Medicines Agency has recommended marketing authorization for Skyclarys to treat Friedreich’s ataxia in patients aged 16 years and older.

Avidity Biosciences Inc. has announced positive data in healthy volunteers from its ongoing phase 1/2 clinical trial of a proprietary monoclonal antibody to treat Duchenne muscular dystrophy mutations amenable to exon 44 skipping.

A company specializing in treatment for neurodegenerative diseases announced positive topline data from a phase 2b clinical trial of a novel, extended release oral fixed-dose therapeutic to treat amyotrophic lateral sclerosis.

The FDA has granted fast track designation for KYV-101, an autologous, fully human CD19 chimeric antigen receptor T-cell product candidate, to treat myasthenia gravis.

The FDA has conferred with BrainStorm Cell Therapeutics Inc. regarding NurOwn, an investigational technology platform developed to treat amyotrophic lateral sclerosis.