From the Journals
Intrathecal therapy was associated with dose-dependent reductions in mutant huntingtin protein, but clinical change will require further study.
PHILADELPHIA – The biomarkers successfully distinguish between healthy controls, patients with premanifest disease,...
Zolgensma is the first gene therapy for the treatment of infantile-onset spinal muscular atrophy in children aged less than 2 years.
Philadelphia – The humanized monoclonal antibody was well tolerated and reduced the risk of disability worsening.
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.