Neuromuscular Disease News

Treatment with inebilizumab was effective in reducing two telltale signs of an attack of neuromyelitis optica spectrum disorder, Bruce A. Cree, MD, PhD, MAS, said in this Healio video.

The U.S. Department of Defense has awarded $11.4 million to a Boston-based biotherapeutics firm to advance its novel oral prodrug of endogenous allopregananolone, LYT-300, to treat Fragile X-associated Tremor Ataxia syndrome.

The FDA has granted breakthrough therapy designation to an investigational antisense nucleotide therapeutic to treat individuals with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.

An analysis of online conversations among health care professionals on social media in June revealed overwhelming support for FDA approvals of therapies for prostate cancer and lymphoma.

Edgewise Therapeutics Inc. announced positive 12-month topline results from the ARCH study evaluating the safety, tolerability and pharmacokinetics of EDG-5506 in adults with Becker muscular dystrophy.

Reata Pharmaceuticals announced that Skyclarys is now commercially available for patients older than 16 years who have Friedreich’s ataxia, a rare degenerative neuromuscular disorder that manifests at a young age.

Horizon Therapeutics announced positive results of MRI data from the phase 3 clinical trial of Uplizna, which showed reduction in formation of subclinical transverse myelitis lesions in patients with neuromyelitis optica spectrum disorder.

The FDA has approved Rystiggo, a subcutaneous infusion therapy to treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibody positive.

The FDA has approved Elevidys, a recombinant gene therapy for pediatric patients with Duchenne muscular dystrophy and confirmed gene mutation who do not have a pre-existing medical reason preventing treatment.

Halozyme Therapeutics announced that argenx received FDA approval for Vyvgart Hytrulo with Enhanze for subcutaneous use in adults with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive.