Amy Burd, PhD, Vice President of Research Strategy at the Leukemia and Lymphoma Society (LLS), discusses her group’s multiarm, multisite collaborative trial which aims to test targeted therapy approaches for improving the generally poor prognosis among patients with AML.
Vanessa Vogel-Farley, Executive Director, Dup15q Alliance discusses Dup15q syndrome, the common name for chromosome 15q11.2-q13.1 duplication syndrome.
The 2017 Emmy-nominated Rare in Common documentary gave people from the rare disease community an opportunity to tell their stories. Today, the storytelling has expanded to the world of audio with the Rare in Common podcast.
Jennifer McNary, Rare Disease Advocate, explains why there is so much interest in Duchenne muscular dystrophy and some of the future considerations for this rare diease.
Barry Ticho, MD, Chief Medical Officer at Stoke Therapeutics, discusses how his company works with Dravet syndrome advocacy groups, including the Dravet Syndrome Foundation (DSF).
Rachelle Dixon, President of the HSAN1E Society discusses the advocacy group she co-founded for hereditary sensory and autonomic neuropathy type 1E (HSAN1E), a progressive disorder of the central and peripheral nervous systems.