Chaperoning The Rare Disease Dance

Emily BurkeThe WEEKLY

PROPERLY FOLDING MISFOLDED DISEASE PROTEINS Amicus Therapeutics (Cranbury, NJ) found itself in the news earlier this month when the FDA agreed to review the company’s new drug application for their investigational therapy to treat Fabry’s disease. The drug under consideration, migalastat, has already been approved by the European Medicines Agency. It belongs to a small, but growing class of therapeutics known … Read More

Unpacking Digital Medicine

Emily BurkeCardiovascular Disease, Diabetes, FDA, Medical Device

CAN APPS PROGRAM BETTER HEALTH? Digital medicine is defined by the field’s pioneer Dr. Eric Tool of the Scripps Translational Science Institute (La Jolla, CA) as “the ability to digitize human beings, by a variety of means (sequencing, sensors, imaging, etc.), fully exploiting our digital infrastructure of ever-increasing bandwidth, connectivity, social networking, the Internet of all things, and health information systems.” This new field is changing the way … Read More

Stopping A Big Problem: Blood Clots

Emily BurkeCocktail Fodder, Drug Approvals, Drug Targets, Mechanism of Action, Small Molecule Drugs

MEDS FOR THINNER BLOOD CAN EQUAL FEWER CLOTS, BUT HOW? The FDA’s recent approval of Portola Pharmaceuticals’ (South San Francisco, CA) new blood thinner drug Bevyxxa paved the way for the prevention of blood clots in patients hospitalized for conditions such as heart failure, stroke, and pulmonary disease. The medical term for blood clot is venous thromboembolism (VTE), but if we take it apart: “venous” means relating … Read More