Neuromuscular Disease News

A combination therapy led to significant dystrophin expression and functional improvement in patients with Duchenne muscular dystrophy amenable to exon 51 skipping, according to new data from a phase 1/2 clinical trial.

Transcranial static magnetic stimulation did not significantly modify ALS disease progression at 6 months compared with sham, but long-term follow up showed an increase in tracheostomy-free survival, data show.

The first patient has been dosed in a phase 3 clinical trial of oral cladribine for the treatment of generalized myasthenia gravis, according to the manufacturer.

The FDA has granted fast-track designation to an investigational tau positron emission tomography diagnostic that can be used for a range of neurodegenerative conditions, according to the manufacturer.

A Georgia-based biotechnology company has secured a U.S. patent for an exosome-based therapy to treat neuroinflammation that occurs in a range of neurodegenerative disease, according to the manufacturer.

The FDA has granted rare pediatric disease designation to an investigational oligonucleotide for the treatment of boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping, according to the manufacturer.

The FDA has granted Regenerative Medicine Advanced Therapy designation to an autologous, fully human CD19 chimeric antigen receptor T-cell therapy candidate, to treat those with progressive myasthenia gravis.

An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular atrophy showed sustained motor function improvements at 48 months, according to the manufacturer.

The FDA has granted Regenerative Medicine Advanced Therapy designation to a Bay Area-based biopharmaceutical company for its autologous, fully human CD19 chimeric antigen receptor to treat those with refractory stiff person syndrome.