Neuromuscular Disease News

The FDA has approved Vyvgart Hytrulo in a once-weekly, 30- to 90-second subcutaneous injection to treat adults with chronic inflammatory demyelinating polyneuropathy, according to the manufacturer.

The FDA has approved an abbreviated new drug application for a generic version of Emflaza to treat Duchenne muscular dystrophy, according to the manufacturer.

Newly released data from the open-label extension of the FIREFISH study confirmed sustained safety and efficacy of Evrysdi in children with type 1 spinal muscular atrophy over 5 years.

Prothena Corp. recently announced it would receive $80 million from Bristol Myers Squibb for an exclusive global license to an investigational therapeutic developed to address protein dysregulation.

The FDA has selected an investigational AAV9 gene therapy for Rett syndrome to participate in a pilot program created to provide a boost to novel drug development for rare diseases, according to a release from the manufacturer.

The European Commission has granted marketing authorization under exceptional circumstances for Qalsody to treat those with amyotrophic lateral sclerosis associated with a mutation in the superoxide dismutase 1 gene.

The FDA has cleared an investigational new drug application for a phase 1/2 clinical trial to examine a novel, once-daily pill for the treatment of individuals with amyotrophic lateral sclerosis, according to the manufacturer.

The FDA has granted fast track designation to a radioactive fluorinated positron emission tomography tracer for imaging tau protein in those with suspected progressive supranuclear palsy, according to the manufacturer.

The Foundation for the NIH has announced the launch of a public-private partnership to advance knowledge of and accelerate drug development for individuals affected by amyotrophic lateral sclerosis.

The FDA has granted orphan drug designation to a small molecule therapeutic for individuals with Fragile X syndrome.