Rare Diseases
From the Journals
Outcomes have improved for PAH in connective tissue disease
Modern treatments and combination approaches may have contributed to overall improvements in survival in patients with pulmonary arterial...
From the Journals
Tocilizumab may improve lung function in early systemic sclerosis
Treatment with tocilizumab may improve lung function in patients with interstitial lung disease associated with systemic sclerosis.
Feature
Dan Kastner wins Crafoord Prize in Polyarthritis
Dr. Kastner received the award for identifying the mechanisms responsible for familial Mediterranean fever, tumor necrosis factor receptor–...
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 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...
Opinion
A 4-year-old presented to our pediatric dermatology clinic for evaluation of asymptomatic "brown spots."
A 4-year-old presents with asymptomatic "brown spots."
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...
Conference Coverage
Birch bark derivative gel found effective for EB, in phase 3 study
"This is the first large clinical trial with placebo of a topical treatment that's worked for this terrible disease."
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...