Neuromuscular Disease News

U.K.-based biotechnology company Pheno Therapeutics announced it has entered into an exclusive worldwide license agreement with Belgian-based biopharmaceutical company UCB to deliver new therapeutics for neurodegenerative disease.

Amylyx Pharmaceuticals Inc. announced an exclusive license and distribution agreement with Neopharm for the commercialization of ALS therapeutic AMX0035 in Israel, Gaza, West Bank and the Palestinian Authority.

LinusBio announced it has raised over $16 million in Series A funding to deliver technology that combines genomics, the environment and biological response to help diagnose and monitor the progression of disorders such as ALS and autism.

Myrtelle Inc. announced updated findings of the open-label phase 1/2 clinical trial of its recombinant adeno-associated virus vector-based investigational gene therapy for Canavan disease, a fatal genetic disorder in children.

Despite the number of new medications produced to treat a range of neurologic diseases, limited utilization is driven by relatively high cost and similar efficacy to less expensive drugs, according to a study published in Neurology.

Treatment with nusinersen led to continued and improved motor function up to 5 years after initial dose in infants with spinal muscular atrophy, according to a poster presented at the International Scientific Congress on Spinal Muscular Atrophy.

The FDA has given priority review to a biologic license application from UCB for rozanolixizumab, a treatment for adults with generalized myasthenia gravis, the biopharmaceutical company announced in a press release.

Athira Pharma Inc. will proceed in 2023 with several clinical trials for treatments for Alzheimer’s disease, Parkinson’s disease and ALS, the biopharmaceutical company announced in a press release.

People with MS who are being treated with rituximab are strongly encouraged to receive mRNA SARS-CoV-2 vaccines and booster doses more than 6 months after their last rituximab infusion, researchers reported in JAMA Network Open.

The FDA has issued a complete response letter to Ipsen for its new drug application for palovarotene, an investigational treatment for the reduction of new abnormal bone formation in people with fibrodysplasia ossificans progressiva.