Rare Diseases
Literature Review
New hope for ALS
A precision-medicine approach to ALS associated with single mutated genes may be within reach.
FDA/CDC
FDA approves new treatment for Dravet syndrome
Children treated with fenfluramine experienced significantly greater reductions in the frequency of convulsive seizures than did their peers who...
Literature Review
First reported U.S. case of COVID-19 linked to Guillain-Barré syndrome
The unique clinical features in the U.S. case are urinary retention secondary to dysautonomia and ocular symptoms of...
Literature Review
Huntington’s disease biomarkers appear 24 years before clinical symptoms
Early signs of neurodegeneration despite normal brain function detected in young adult carriers of the Huntington’s disease gene.
Conference Coverage
New research confirms the efficacy and safety of onasemnogene abeparvovec for SMA
Three investigations indicate that the treatment allows children, including those who are presymptomatic, to achieve and maintain motor milestones...
News
Two rare neurologic conditions linked to COVID-19
Two variants of Guillain-Barré syndrome—Miller Fisher syndrome and polyneuritis cranialis—have been linked with COVID-19 infection.
FDA/CDC
FDA approves Koselugo for pediatric neurofibromatosis treatment
News
First case of COVID-19 presenting as Guillain-Barré reported
A case report from China suggests a possible association between GBS and SARS-CoV-2 infection.
Medical Education Library
Rare Neurological Disease Special Report
FDA/CDC
Upcoming vaccine may offset surge in polio subtypes
Response to polio outbreaks involves fast-tracking a modified oral vaccine.
Conference Coverage
Inebilizumab benefits patients with NMOSD
WEST PALM BEACH, FLA. – The treatment reduces the risk of attacks, the risk of disability worsening, and the number...