Rare Diseases and the Challenges Ahead

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...

Biomarkers for Type 1 Gaucher Disease | Video

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...

BMS Sells Two Orphan Drugs to Biogen and Roche

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...