Upcoming FDA Dates for Rare Disease and Orphan Drugs

June 17:  PDUFA date for Johnson & Johnson’s DARZALEX (Daratumumab) + pomalidomide and dexamethasone for Relapsed or refractory multiple myeloma June 24:  Preliminary data due at European Hematology Association (Phase 2a initiated June 2016) for Global Blood...

How I learned to See Through The Eyes of My Sons

When Kristin Smedley learned that her first child was born with a gene mutation that causes blindness, she believed the life she dreamt for her son was over. Smedley shares her very personal story of how she learned to see the world differently through the eyes of her...

FDA Approves Novo Nordisk Treatment for Hemophilia

The FDA has approved Novo Nordisk’s Rebinyn coagulation treatment for the treatment of adults and children with hemophilia B. Rebinyn is indicated for treatment and control of bleeding episodes and the perioperative management of bleeding in adults and children with...