by Peter Ciszewski | Oct 19, 2018
The FDA granted Olaparib (Lynparza) an orphan drug designation by for the treatment of patients with pancreatic cancer, Pancreatic cancer is a rare, life-threatening disease that accounts for about 3% of all cancers in the US.Due to the late onset of symptoms,...
by Peter Ciszewski | Oct 18, 2018
Katherine Stueland, Chief Commercial Officer at Invitae, discusses how genetic testing companies partner with drug developers, including her company’s work with Alnylam. Invitae is a genetic information company bringing genetic information into mainstream...
by Peter Ciszewski | Oct 17, 2018
Jennifer McNary is a consultant, speaker, and rare disease advocate. She is also a mother of two boys with Duchenne muscular dystrophy (DMD). In this video, Jennifer discusses her two boys, Max and Austin, and their journey with DMD as well as the some of the...
by Peter Ciszewski | Oct 12, 2018
Len Walt, Vice President, Medical Affairs, North America discusses his company’s focus on rare diseases, specificially MPS IIIA or Sanfilippo A syndrome. MPS IIIA is a progressive, life-threatening and rare inherited metabolic disorder affecting children...
by Peter Ciszewski | Oct 10, 2018
The FDA approved Revcovi (elapegademase-lvlr) injection in the United States, for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. “We are gratified by the FDA’s timely recognition of...
