by Peter Ciszewski | Oct 31, 2022
Olaf Bodamer, MD, PhD, Associate Chief of Genetics and Genomics at Boston Children’s Hospital, discusses how the National Organization for Rare Disorders’ (NORD) Rare Disease Centers of Excellence may help rare disease patients receive the best care...
by Peter Ciszewski | Oct 26, 2022
Nathalie Dubois-Stringfellow, PhD, Chief Development Officer at Sangamo Therapeutics, discussed the promising results from their ongoing gene therapy trial to treat Fabry disease. Fabry disease is a rare X-linked lysosomal storage disorder that results in the...
by Peter Ciszewski | Oct 22, 2022
Ronald Martell, President, and CEO of Jasper Therapeutics, discusses the positive data from the ongoing phase 1/2 study testing JSP191 as a conditioning agent in the treatment of Fanconi anemia. These data were presented at the annual conference of the Inborn...
by Peter Ciszewski | Oct 19, 2022
Ricardo Dolmetsch, PhD, President of Research and Development at uniQure, discusses what he thinks physicians should know about gene therapy. Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic starting at around...
by Peter Ciszewski | Oct 18, 2022
Evan S. Dellon, MD, MPH, Gastroenterologist, and Professor of Medicine and Adjunct Professor of Epidemiology at the University of North Carolina Chapel Hill, explains two things that physicians should know about eosinophilic esophagitis (EoE). EoE is a chronic,...
