by Peter Ciszewski | Jun 7, 2017
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...
by Peter Ciszewski | Jun 6, 2017
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...
by Peter Ciszewski | Jun 4, 2017
ArQule announced that data from a phase 1/2 trial with fibroblast growth factor receptor (FGFR) inhibitor, ARQ 087, presented at ASCO demonstrate a meaningful clinical benefit to intrahepatic cholangiocarcinoma (iCCA) patients harboring FGFR2 fusions. The data show a...
by Peter Ciszewski | Jun 1, 2017
Dr. Pramod Mistry provides an update of the ENGAGE trial, which evaluating Cerdelga (eliglustat) for treatment-naïve patients with type 1 Gaucher disease. Cerdelga is an oral substrate reduction therapy and is approved as a first-line treatment for adults with type 1...
by Peter Ciszewski | May 31, 2017
There has been significant pharmaceutical and biotech R&D activity in Duchenne muscular dystrophy (DMD) over the last five years, and that activity has only increased with the approval of Sarepta’s Exondys 51 (eteplirsen) last September. Exondys 51 is...
