by Peter Ciszewski | Mar 28, 2019
Ron Cooper, President and CEO of Albireo, provides and overview of progressive familial intrahepatic cholestasis (PFIC), a rare genetic condition that affects the liver. PFIC is estimated to affect between one in every 50,000 to 100,000 children born worldwide...
by Peter Ciszewski | Mar 27, 2019
Stefan Weber, CEO of Newron Pharmaceuticals, provides an update on the “Sarizotan Treatment of Apneas in Rett Syndrome” (STARS) study, a pivotal clinical study performed in study centers in the US, Europe, Asia, and Australia to evaluate the efficacy, safety...
by Peter Ciszewski | Mar 26, 2019
Amanda Haidet-Phillips, PhD, Scientific Portfolio Director at the Muscular Dystrophy Association, discusses her organization and its successes funding research to accelerate treatments and cures across the full spectrum of neuromuscular diseases. Dr....
by Peter Ciszewski | Mar 25, 2019
Hartmann Wellhoefer, MD, Vice President, Medical Affairs, Rare Disease and Internal Medicine, Global R&D at Takeda sat down with CheckRare to discuss the 15th Annual WORLDSymposium 2019 to share his insights about this year’s conference including key...
by Peter Ciszewski | Mar 25, 2019
Benjamin Forred, Translational Research Project Manager at Sanford Health in Sioux Falls, South Dakota, discusses Rare Disease Day and his organization which is one of the largest health systems in the United States, including research into rare...
