News from the FDA/CDC
The central nervous system depressant was approved for the treatment of cataplexy and excessive daytime sleepiness.
WASHINGTON – Most patients with opsoclonus myoclonus syndrome experience ataxia, among multiple other symptoms.
Gene-Replacement Therapy for SMA1 May Necessitate New Rating Measures to Capture Patients’ Motor Function Gains
CHICAGO—The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) was designed to track motor function...
From the Journals
One patient fully recovered and a second improved but had speech and mobility problems.
Inotersen inhibits the production of amyloid in patients with the rare disorder.
HILTON HEAD, SC—Vasculitis is a general term for a group of uncommon diseases involving inflammation of blood vessels,...
HILTON HEAD, SC—Behavioral variant frontotemporal dementia (bvFTD), a clinically and pathologically heterogenous...
On September 24, 2018, the FDA announced that it awarded 12 new clinical trial research grants totaling more...
A new system that incorporates clinical and serologic data may help classify idiopathic inflammatory myopathies,...