Rare Diseases
Article
Examining the Role of Caregivers at NINR Caregiving Summit
Article
FDA Commissioner to Present Keynote Address at NORD Summit
Article
Preview Agenda for NORD’s 2017 Rare Diseases and Orphan Products Breakthrough Summit
Conference Coverage
Bone marrow transplantation for epidermolysis bullosa continues to evolve
Today, bone marrow transplantation is the most widely used stem cell therapy for patients with severe EB.
From the Journals
Blood markers can detect early pancreatic cancer
A test in development is recommended for those at high risk and those newly diagnosed with diabetes.
Conference Coverage
TRK inhibitor shows ‘striking’ activity, durability across diverse adult and pediatric cancers
An analysis of three trials involving 55 patients with advanced cancer harboring TRK fusions who were treated with larotrectinib showed an overall...
News
FDA approves Rebinyn for hemophilia B treatment
Rebinyn is intended to control and treat bleeding episodes, as well as provide perioperative management of bleeding.