by Peter Ciszewski | May 13, 2019
Vanessa Vogel-Farley, Executive Director of the Dup15q Alliance, discusses research in this rare disease, including the ARCADE trial, a phase 2 open-label pilot study. OV935 may be effective in controlling seizures. Four phase I studies successfully evaluated...
by Peter Ciszewski | May 12, 2019
Anne R. Pariser, MD, Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences at the National Institutes of Health discusses rare disease research.
by Peter Ciszewski | May 10, 2019
Mark Pykett, PhD, Chief Scientific Officer at PTC Therapeutics, discusses his company’s product for Duchenne muscular dystrophy, Translarna.
by Peter Ciszewski | May 8, 2019
Debra Miller, founder of CureDuchenne, discusses some of the decisions that Duchenne muscular dystrophy (DMD) families and patients need to consider when considering a clinical trial. With so much research activity in DMD, organizations like CureDuchenne can...
by Peter Ciszewski | May 7, 2019
Scott Schobel, MD, MSc, Associate Group Medical Director and Clinical Science Leader for the IONIS/Roche HTT Rx program at Roche/Genentech Neuroscience discusses Huntington’s disease and the GENERATION HD1 trial (Global EvaluatioN of Efficacy and Safety...
