by Peter Ciszewski | Oct 1, 2020
Jörn-Sven Kühl, MD, Department of Pediatric Oncology, Hematology and Hemostaseology at University Hospital Leipzig, discusses long-term results from the Phase 2/3 Starbeam study and data from the Phase 3 ALD-104 study. Bluebird bio, Inc. recently presented data...
by Peter Ciszewski | Sep 30, 2020
Erik Harris, Executive Vice President and Chief Commercial Officer at Ultragenyx Pharmaceutical Inc. discusses how the COVID-19 pandemic has impacted the company. According to Mr. Harris, Ultragenyx has always worked closely with patients and pharmacies to...
by Peter Ciszewski | Sep 27, 2020
Kelly Heger, Founder of the AADC Family Network, provides an overview of aromatic L-amino acid decarboxylase (AADC) deficiency and her family’s diagnostic journey with the disorder. Kelly is the mother and caregiver of Jillian Heger, who lives with AADC...
by Peter Ciszewski | Sep 24, 2020
Orna Staretz-Chacham, MD, of the Ben-Gurion University of the Negev, Israel talks about the impact the COVID-19 pandemic has had on lysosomal storage disease patients in Israel. This presentation was part of the live GRIDS 2020-Virtual Summit held July 1, 2020...
by Peter Ciszewski | Sep 23, 2020
Emil Kakkis, MD, President and Founder of Ultragenyx Pharmaceutical Inc., explains how the Orphan Drug Act of 1983 may have been the catalyst to begin developing drugs for rare diseases, other factors are now in place to make orphan drug development a viable...
