Rare Diseases
News
NORD and SLS Community Launch Registry for Patients and Researchers
News
NORD Announces Rare Disease Research Funding Opportunities
Article
World Federation of Hemophilia Seeks Proposals
Article
Progeria Research Foundation is Accepting Applications for Grants
Article
Congenital Hyperinsulinism International Issues Statement of Support for New Recommendations
Conference Coverage
DDW: Study finds pancreatic cancer misdiagnosis rate at 31%
Key clinical point: Pancreatic cancer is often misdiagnosed, and should be considered in patients with vague or atypical symptoms who are referred...
News
FDA approves Orkambi for cystic fibrosis
News
Afamelanotide improves erythropoietic protoporphyria
Key clinical point: Afamelanotide is the first effective treatment for erythropoietic protoporphyria, an extremely painful photodermatosis that...
Applied Evidence
Monoclonal gammopathy of undetermined significance: Using risk stratification to guide follow-up
Varying combinations of 3 measurable factors determine a patient’s risk of progressing toward multiple myeloma and influence monitoring decisions...
Photo Rounds
Scarring alopecia in a woman with psoriasis
Was this patient’s plaque psoriasis causing her progressive hair loss—or was it something else?
News
Researchers pin down possible cause, source for narcolepsy associated with flu vaccine
Key clinical point:Nucleoprotein antibodies possibly explain influenza vaccine associated narcolepsy. Major finding: Patients with a history of...