Neuromuscular Disease News

Small fiber neuropathy, a pain-inducing condition that resembles and overlaps with fibromyalgia, may be significantly under-diagnosed in the U.S., according to a presenter at the 2021 Congress of Clinical Rheumatology-West.

BridgeBio Pharma Inc. announced it has received FDA fast track designation for BBP-418 as a treatment option for limb-girdle muscular dystrophy type 2i.

Increased levels of neurofilament light in plasma samples correlated with a greater risk for ALS, according to findings from an analysis of plasma samples obtained within 5 years of an ALS diagnosis that was published in Neurology.

Researchers determined deltoid muscle strength to be the top clinical outcome measure when it comes to capturing oculopharyngeal muscular dystrophy progression, according to findings published in Neurology.

Nearly half of individuals in a study about activities of daily living among more than 1,000 patients with myasthenia gravis reported disease symptoms associated with an “unsatisfactory” state, according to findings published in Neurology.

Patients with myasthenia gravis — particularly those post-thymectomy — demonstrate an increased odds for having systemic lupus erythematosus, according to a speaker at the 2021 North American Young Rheumatology Investigator Forum.

The FDA today approved Nexviazyme, an IV infusion, for the treatment of late-onset Pompe disease among patients aged 1 year and older, according to a press release.

The presence of pain in children with cerebral palsy can make motor skill activities even more difficult, according to a research letter published in JAMA Network Open.

Better screening protocols and new, potentially more effective treatments have created “an opportune time” for the implementation of newborn screening for Duchenne muscular dystrophy, according to a viewpoint published in JAMA Neurology.

The FDA granted orphan drug designation to ALS001, an autologous, expanded T-cell therapy under development by Coya Therapeutics for treating ALS, according to a press release.