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...
by Peter Ciszewski | May 30, 2017
An influential think tank funded by insurance companies and pharmacy benefit managers called the Institute for Clinical and Economic Review (ICER) is holding a policy summit in Washington, DC on Wednesday (May 31) to discuss “methods for assessing the value of...
by Peter Ciszewski | May 23, 2017
The use of biomarkers can provide valuable insight into the molecular pathogenesis of lysosomal storage diseases such as Gaucher disease and Fabry disease. Dr. Pramod Mistry discusses biomarkers as they pertain to patients with type 1 Gaucher disease. In Gaucher...
by Peter Ciszewski | May 13, 2017
Bristol-Myers Squibb Company announced that it has entered into two separate agreements to license BMS-986168, an anti-eTau compound in development for Progressive Supranuclear Palsy (PSP), to Biogen, and BMS-986089, an anti-myostatin adnectin in development for...
by Peter Ciszewski | May 13, 2017
by: CheckRare Staff Bayer announced that the U.S. Food and Drug Administration (FDA) approved Stivarga (regorafenib) tablets for the second-line treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with...
