Neuromuscular Disease News

The European Commission has authorized Skyclarys to treat Friedreich’s ataxia in adults and adolescents aged 16 years and older.

Positive associations were found between smoking and risk of developing ALS, with the strongest correlation found among those who actively smoked, according to research from Tobacco Induced Diseases.

The European Commission has approved Hyqvia as maintenance therapy for individuals of all ages with chronic inflammatory demyelinating polyneuropathy.

The FDA has approved Gammagard Liquid as an IV immunoglobulin therapy to improve neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy.

The FDA has granted orphan drug designation to an investigational therapeutic to treat myotonic dystrophy type 1, the most common form of muscular dystrophy in adults, according to its manufacturer.

A Houston-area biotech company will expand indications of its investigational and proprietary biologic combination therapy beyond amyotrophic lateral sclerosis to include frontotemporal dementia and Parkinson’s disease.

The FDA has cleared an investigational new drug application for an unmodified neural-derived exosome to treat those with acute ischemic stroke, according to its manufacturer.

The FDA has approved Takeda Pharmaceutical’s Hyqvia, coformulated with Halozyme's Enhanze drug delivery technology, to treat chronic inflammatory demyelinating polyneuropathy in adults.

A clinical-stage genetic medicine firm has announced $60 million in Series B financing to support a phase 1/2 clinical trial and advance its lead candidate to treat spinocerebellar ataxia types 1 and 3 as well as Huntington’s disease.

The European Medicines Agency has approved enrollment for a phase 1/2a biomarker study to examine a once-daily, oral, brain penetrant mitochondrial stimulator to treat a range of neurological and neuromuscular conditions.