Neuromuscular Disease News

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.

In patients with neuromyelitis optica spectrum disorder, transition from rituximab to inebilizumab was linked to suppression of attacks over a mean 19 months of treatment, according to research published in Frontiers in Neurology.

DENVER — A combination therapy of ciprofloxacin and celecoxib demonstrated a favorable safety profile for those with amyotrophic lateral sclerosis, Merit Cudkowicz, MD, MSc, said in this Healio video.

The FDA has granted breakthrough therapy designation to a proprietary oligonucleotide conjugate for the treatment of myotonic dystrophy type 1, according to the manufacturer.

DENVER — Experiences and concerns about treatment, diagnosis, care, cost and quality of life among Black and Hispanic patients with myasthenia gravis were more negative compared with their Asian and white counterparts, according to research.