Neuromuscular Disease News

A United Kingdom-based biotech company has announced a global licensing agreement with a California pharma firm to research and develop drug candidates targeting two different ion channel targets associated with neurological disorders.

A nonprofit focused on treatments and cures for Charcot-Marie-Tooth disease will partner with a biotechnology firm to advance a novel intrathecal miRNA therapy to address CMT type 1a in adults with the condition.

Nido Biosciences has announced positive results from a phase 1 study of its novel, small molecule therapeutic to treat spinal and bulbar muscular atrophy in healthy male participants.

The FDA has approved a 40 mg/mL oral suspension of Agamree to treat Duchenne muscular dystrophy in patients aged 2 years and older, according to a release from the manufacturer.

A new set of evidence-based guidelines for ALS genetic testing are focused on improving knowledge among all stakeholders, according to a report in the Annals of Clinical and Translational Neurology.

The FDA has approved UCB’s Zilbrysq for the treatment of generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive.

A Southern California biopharmaceutical company has announced positive data from a phase 1/2 clinical trial of its proprietary monoclonal antibody, AOC 1001, for those with myotonic dystrophy type 1.

Genentech has announced positive results from its primary analysis of an ongoing study assessing safety and efficacy of Evrysdi in babies with pre-symptomatic spinal muscular atrophy.

A Bay Area pharmaceutical firm has announced positive data from a phase 3 clinical trial evaluating Chenodal tablets to treat adults with cerebrotendinous xanthomatosis, an autosomal, recessive, progressive genetic disorder.

Regeneron Pharmaceuticals Inc. and Intellia Therapeutics Inc. have announced an expanded research collaboration intended to develop additional CRISPR-based gene editing therapies focused on neurological and muscular diseases.