Rare Diseases
Conference Coverage
CBD for LGS: Fewer seizures, but thrombocytopenia risk
Cannabidiol reduces seizures in Lennox Gastaut syndrome out to 3 years, but has been linked to thrombocytopenia in children also on valproic acid...
Conference Coverage
NMOSD challenges in children
Neuroimmunologists discuss key diagnosis and treatment strategies.
Literature Review
Drug combo slows functional decline in ALS
Patients who received the drug combination “retained higher levels of physical function over 6 months compared with those who received placebo.”...
From the Journals
TNF inhibitors linked to inflammatory CNS events
The authors do not caution “against using these therapies in appropriate patients,” and it is unclear whether this association “represents de novo...
Literature Review
COVID-19 linked to development of myasthenia gravis
Italian researchers report three cases of myasthenia gravis onset after COVID-19 infection.
FDA/CDC
FDA approves viltolarsen (Viltepso) for Duchenne muscular dystrophy
The approval marks the second drug therapy for Duchenne muscular dystrophy.
FDA/CDC
FDA approves first oral treatment for spinal muscular atrophy
The oral treatment provided a “meaningful difference from the natural progression of the disease.”
From the Journals
Psychiatrists report rare case of woman who thinks she’s a chicken
"It's a good thing that we psychiatrists are aware of the fact that clinical zoanthropy exists and may require additional research," said Dr....
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...