PHILADELPHIA – In patients with amyotrophic lateral sclerosis caused by mutations in the SOD1 gene, an investigational treatment reduced SOD1...
The approval is the first for a LEMS treatment specifically for pediatric patients.
PHILADELPHIA – Experimental treatment may benefit adolescents and adults with Angelman syndrome.
An analysis suggests the possibility of using a minimally invasive biomarker to guide clinical care and improve clinical trial design.
BALTIMORE – Some Zika-exposed children showed poorer mobility and social cognitive scores as they approached 18 months even though their fetal...
A higher risk of ALS and an earlier age of onset was found among professional soccer players.
A population-based study found more than 10% of European adults carried CAG repeats in the pathogenic range, despite lacking signs of disease.
Data from a phase 2 study expand on previous findings and suggest that trofinetide can alleviate core symptoms of the neurodevelopmental disorder...
Thinking polymyositis? Look closely for IMNM, overlap with other rheumatologic conditions, antisynthetase syndrome, inclusion body myositis, and...
Patients with generalized nonthymomatous myasthenia gravis who undergo thymectomy may have better long-term clinical outcomes and require less...