Rare Diseases
Article
Pitt Hopkins Scientific Symposium to Take Place in November
News
Midostaurin cut organ damage in systemic mastocytosis
Key clinical point: Midostaurin helped to resolve organ damage related to mastocytosis. Major finding: In all, 45% of patients had complete...
Conference Coverage
Updated Behçet’s disease recommendations expand biologic treatment
Conference Coverage
Edaravone slows progression of ALS when started early on
Key clinical point: When added to standard of care, edaravone slows loss of function in patients with early-stage ALS. Major finding: Compared...
Conference Coverage
Giant cell arteritis survival doubled in the past 2 decades
Key clinical point: The survival of patients with giant cell arteritis has more than doubled in the past 20 years. Major finding: Comparing two...
Conference Coverage
Enlist appropriate psychological consults for gender dysphoria
ORLANDO – There is now an awareness of a spectrum of gender identity and an affirmation of the right of individuals to express that identity as...
News
Ocaliva approved for primary biliary cholangitis
Photo Rounds
Large plaques on a baby boy
This newborn’s prenatal lab work and delivery were normal. So, too, were his Apgar scores and neurologic exam. So why was he covered in brown and...
Case Reports
Darkened skin, vomiting, and salt cravings in a teenager • Dx?
► Headache, dizziness, lethargy, weakness► Vomiting twice daily for 3 years► Darkened skin► Salt cravings
News
Materials help families find support for children with serious illnesses
News
Stevens-Johnson syndrome, TEN not as rare as thought
Stevens-Johnson syndrome and toxic epidermal necrolysis are not quite as rare as previously thought.