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.
Feature
CDER chief reflects on advances in rare diseases
“People always call me fearless, but I feel like I just state the facts,” Dr. Janet Woodcock said.
News from the FDA/CDC
Golimumab approval extended to polyarticular-course JIA and juvenile PsA
The expanded approval to include pediatric indications came as part of a review of a clinical trial mandated by the Pediatric Research Equity Act...
News from the FDA/CDC
FDA adds polyarticular-course JIA to approved indications for tofacitinib
The approval is the fourth indication in the United States for tofacitinib after approvals for treatment of adults with rheumatoid arthritis,...
From the Journals
Canakinumab (Ilaris) tapering tested in systemic JIA trial
Reducing or extending the frequency of canakinumab over 6 months enabled children to maintain complete clinical remission and some to stop...
News
FDA approves viltolarsen (Viltepso) for Duchenne muscular dystrophy
The approval marks the second drug therapy for Duchenne muscular dystrophy.
News
FDA approves first oral treatment for spinal muscular atrophy
The oral treatment provided a “meaningful difference from the natural progression of the disease.”
News
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...
News
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...
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...