by Peter Ciszewski | Dec 13, 2019
The U.S. Food and Drug Administration has approved Vyondys 53 (golodirsen) to treat patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Golodirsen is an antisense oligonucleotide that can ‘skip’ over aspects of the RNA to make person’s with...
by Peter Ciszewski | Dec 12, 2019
David Roth, MD of Syros Pharmaceuticals describes his company’s gene control methodology to target genetic conditions in a unique way. Instead of the more traditional gene therapies that are the focus of many studies, Syros investigates and identifies...
by Peter Ciszewski | Dec 11, 2019
Jonathan C. Roberts, MD, of the Bleeding & Clotting Disorders Institute, Peoria, IL talks about a recent claims data for children and adolescents with Von Willebrand disease. The study looked at medical insurance claims for persons Von Willebrand disease...
by Peter Ciszewski | Dec 10, 2019
Peter Voorhees, MD of the Levine Cancer Institute in Charlotte, NC provides an update on the Griffin Study, a Phase 2 clinical trial comparing subcutaneous daratumumab in combination with standard-of-care regimens for newly diagnosed multiple myeloma....
by Peter Ciszewski | Dec 9, 2019
Jennifer Crombie, MD, of the Department of Medical Oncology at Dana-Farber Cancer Institute in Boston, MA discusses the results of a phase I study testing the combination of duvelisib and venetoclax in patients with relapsed or refractory chronic lymphocytic...
