by Peter Ciszewski | Nov 5, 2018
Richard A. Basile,Co-Founder and CEO of BioPontis Alliance for Rare Diseases, discusses the challenges of developing drugs for rare diseases. BioPontis is an international nonprofit organization whose mission is to advance promising science into potential...
by Peter Ciszewski | Nov 3, 2018
Katherine Stueland, Chief Commercial Officer at Invitae, discusses how diagnostic companies work with biotech companies. Katherine also discusses the role of genetic companies in assisting patients and physicians in diagnosing rare diseases. Invitae is a...
by Peter Ciszewski | Nov 2, 2018
In the United States, an estimated 2.5 to 3 million people have a gene variant associated sickle cell trait (SCT) and most never have related health complications. Researchers have now identified three health conditions for which people with SCT are at increased risk....
by Peter Ciszewski | Nov 2, 2018
Jennifer McNary, rare disease advocate, looks back at the Sarepta AdComm for Exondys 51 in 2016. She also discusses the role of the patient advocate. Duchenne muscular dystrophy occurs in one out of every 3,600 male infants, and it’s even rarer in girls....
by Peter Ciszewski | Oct 29, 2018
Len Walt, Vice President, Head of Medical Affairs, SOBI in North America, discusses his company’s activities in Hemophagocytic lymphohistiocytosis (HLH), a rare condition in which the body makes too many activated immune cells (macrophages and...
