Neuromuscular Disease News

On Dec. 8, Grammy award-winning artist Celine Dion announced that she would be rescheduling her 2023 tour dates after being diagnosed with stiff-person syndrome, a progressive neurological disorder.

The use of machine learning models identified three distinct subtypes of Parkinson’s disease, which “could have immediate implications” in detecting clinical outcomes, researchers reported in NPJ Parkinson’s Disease.

The annual incidence of Parkinson’s disease among adults aged 65 years and older is 50% higher than previous estimates of 60,000 diagnoses annually, researchers reported in NPJ Parkinson’s Disease.

Avidity Biosciences Inc. announced positive data from a phase 1/2 trial, which successfully delivered RNA into muscle for the first time in a study that aimed to treat patients with myotonic dystrophy, the company said in a press release.

Biogen announced that the European Medicines Agency has accepted the marketing authorization application for tofersen, an investigational drug for the treatment of superoxide dismutase 1 ALS.

Clinical-stage biopharmaceutical company Clene Inc. announced proof-of-concept evidence of potential neuroprotective effects of CNM-Au8, an oral therapeutic for patients with stable relapsing-remitting MS.

Nearly 90% of patients with relapsing-remitting MS in a multicenter study in Germany preferred subcutaneous administration of Tysabri rather than IV, citing shorter duration of delivery and convenience, per a presentation at ECTRIMS 2022.

Preclinical biotech company Eikonoklastes Therapeutics and gene therapy manufacturer Forge Biologics announced a partnership that will advance Eikonoklastes’ ET-101 gene therapy into clinical trials for patients with ALS.

Results from the EVOLVE-MS-1 study show Vumerity was effective and well-tolerated over 96 weeks in patients with relapsing-remitting MS compared with dimethyl fumarate, according to a presentation at ECTRIMS 2022.

Lysogene has announced topline results from its phase 2/3 study evaluating LYS-SAF302, an investigational gene therapy for the treatment of mucopolysaccharidosis type IIIA, or Sanfilippo syndrome.