by Peter Ciszewski | Sep 15, 2022
Ruchira Glaser, MD, MS, Senior Vice President, Therapeutic Area Head of Rare Disease, Autoimmune & Cardiovascular at Moderna, discusses the phase 1/2 Paramount study evaluating investigational mRNA-3927 in participants with propionic acidemia (PA). PA is a rare...
by Peter Ciszewski | Sep 14, 2022
Ricardo Dolmetsch, PhD, President of Research and Development at uniQure, gives an overview of Huntington disease. As Dr. Dolmetsch explains, Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic starting at...
by Peter Ciszewski | Sep 12, 2022
Joslyn Crowe, executive director of the National Niemann-Pick Disease Foundation (NNPDF) talks about the recent approval of olipudase alfa to treat pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD). ASMD is an autosomal recessive genetic...
by Peter Ciszewski | Sep 9, 2022
Gregory Davis, PhD, Vice President of Genome Engineering at Sangamo Therapeutics, gives an overview of zinc finger proteins which play a crucial part in Sangamo’s proprietary zinc finger platform. As Dr. Davis explains, zinc finger proteins are naturally...
by Peter Ciszewski | Sep 7, 2022
Barry S. Ticho, MD, PhD, Chief Medical Officer at Stoke Therapeutics, gives a detailed overview of Dravet syndrome. As Dr. Ticho explains, Dravet syndrome is a rare neurological condition that usually appears during the first year of life as frequent febrile...
