Neuromuscular Disease News

Health Canada has approved Daybue to treat Rett syndrome in patients aged 2 years and older, according to manufacturer Acadia Pharmaceuticals.

Bluebird Bio’s lentiviral vector gene therapy Skysona met its primary efficacy endpoint of survival without major functional disability in 81% of boys with early cerebral adrenoleukodystrophy, a recent analysis showed.

Topline results from the phase 3 SAPPHIRE clinical trial showed that an investigational muscle-targeted therapy improved motor function in patients with spinal muscular atrophy compared with placebo as early as week 8.

ORLANDO, Fla. — Managing dystonic cerebral palsy in younger populations presents unique challenges, which may be addressed with direct cerebellar implantation of a deep brain stimulation device, according to a presenter.

ORLANDO, Fla. — An algorithm for standardizing myasthenia gravis diagnosis during telehealth is feasible, but it requires additional testing and validation, according to researchers.

ORLANDO, Fla. — Cell-based therapies and enzyme inhibitors currently in development are likely to become the next-generation treatments for ambulatory and non-ambulatory forms of muscular dystrophy, according to a speaker.

Topline data from an ongoing clinical trial of a higher dose regimen of Spinraza in treatment-naive, symptomatic infants with spinal muscular atrophy showed significantly improved motor function at 6 months.

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.