Rare Diseases
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...
Conference Coverage
Eculizumab reduces relapse-related hospitalizations in patients with NMOSD
WEST PALM BEACH, FLA. – The monoclonal antibody may reduce health care resource utilization in this patient...
Literature Review
Zilucoplan improved efficacy outcomes in myasthenia gravis
The complement C5 inhibitor had a similar magnitude of clinical effect as that seen in eculizumab studies.
News from the FDA/CDC
Zika virus: Birth defects rose fourfold in U.S. hardest-hit areas
U.S. jurisdictions without local transmission of the virus saw no increase in infants born with brain abnormalities and/or microcephaly or with...