Rare Diseases
From the Journals
Data call for biologics trials in undertreated juvenile arthritis subtype
Many children with enthesitis-related arthritis have significant disease burden despite treatment with biologics currently approved for adults...
News
FDA clears device to remove dead pancreatic tissue
Conference Coverage
Childhood Hodgkin survivors have neurocognitive impairment
News
FDA clears first drug for rare genetic causes of severe obesity
Setmelanotide (Imcivree) is indicated for weight management in adults and children as young as 6 years with severe obesity driven by genetic...
Conference Coverage
GLIMMER of hope for itch in primary biliary cholangitis
Moderate to severe pruritus “affects patients’ quality of life and is a huge burden for them.”
Conference Coverage
GLIMMER of hope for itch in primary biliary cholangitis
Moderate to severe pruritus “affects patients’ quality of life and is a huge burden for them.”
Conference Coverage
Higher cardiovascular risks in Kawasaki disease persist 10-plus years
Researchers evaluated the incidence of myocardial infarction, stroke, death, and more in a follow-up study of Kawasaki disease survivors, compared...
From the Journals
ASSET extension supports abatacept as treatment for diffuse cutaneous systemic sclerosis
“Exploratory outcome measures during the [6-month] open-label extension, including the composite ACR CRISS score,...
News from the FDA/CDC
Systemic sclerosis patients share their perspectives and needs in treatment trials
FDA officials and clinical researchers heard about the impact that different symptoms have on patients’ daily lives in order to design better...
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...