Rare Diseases
Latest News
Oral drug seen preventing angioedema attacks
A novel agent led to a nearly 75% reduction in monthly attacks among people with a rare genetic disorder.
News
FDA approves Epidiolex for Lennox-Gastaut syndrome and Dravet syndrome
The approval, the first for a marijuana-derived pharmaceutical product, falls in line with the unanimous recommendation of an FDA advisory panel...
News
FDA approves rituximab for treating pemphigus vulgaris
Approval was based on a prospective, multicenter, open-label, randomized study conducted in France.
Conference Coverage
Pemphigus remission rate tops 80% with rituximab
ORLANDO – “Rituximab is quickly emerging as frontline therapy” for pemphigus.
Conference Coverage
Malignant pleural mesothelioma guidelines often are ignored
SAN DIEGO – As many as 32% of patients received no treatment, but their median months of survival weren’t much different than others.