Neuromuscular Disease News

The FDA has approved Skyclarys, Reata Pharmaceuticals’ oral, once-daily medication to treat Friedreich’s ataxia in people aged 16 years and older.

Teva Pharmaceuticals announced FDA approval of Austedo XR, a once-daily, extended-release formulation for adults with tardive dyskinesia and chorea associated with Huntington’s disease.

Ashvattha Therapeutics announced that the first patient has been enrolled in a phase 1/2 study to evaluate the ability of an imaging agent to cross the blood-brain barrier and target key markers of neuroinflammation.

Nova Mentis Life Science Corp. has received an exemption under Section 56 of the Health Canada Controlled Drugs and Substances Act to advance a phase 2a clinical trial examining oral microdose psilocybin therapy for fragile X syndrome.

The FDA has granted orphan drug designation to Avidity Biosciences Inc. for AOC 1020, its novel RNA therapeutic to treat facioscapulohumeral muscular dystrophy.

Athira Pharma Inc. revealed its list of planned clinical trials for 2023, which include development of therapeutics for Alzheimer’s disease, Parkinson’s disease and ALS.

Although well-tolerated with few adverse events, treatment with leriglitazone did not slow disease progression in men with adrenomyeloneuropathy compared with placebo, per a study published in Lancet Neurology.

Researchers recently found that the pharmacokinetic properties of vesicular monoamine transporter 2 inhibitors may play a role in the treatment of tardive dyskinesia.

Cycle Pharmaceuticals announced that the FDA has approved its Tascenso ODT, a bioequivalent to Gilenya, or generic fingolimod, for patients with relapsing forms of MS.

The FDA has granted fast track designation to Avidity Biosciences Inc. for its novel RNA therapeutic AOC 1020, an antibody oligonucleotide conjugate being studied for the treatment of facioscapulohumeral muscular dystrophy.