by Peter Ciszewski | Oct 2, 2020
Peter Saltonstall, President and Chief Executive Officer of the National Organization for Rare Disorders (NORD), discusses the upcoming Virtual Rare Summit being held October 8-9. For over 30 years, NORD has been providing services for rare disease patients and...
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...
