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...
by Peter Ciszewski | May 6, 2019
Prof. Arndt Rolfs, MD, Chief Executive Officer at Centogene discuses his company’s role in identiying rare diseases. Centogene is focused on transforming clinical, genetic, and biochemical data into medical solutions for patients in international...
by Peter Ciszewski | May 6, 2019
Peter Saltonstall, President and Chief Executive Officer of the National Organization for Rare Disorders (NORD), discusses how our current drug payer system has not kept up with the science of medicine. We need to understand as a society how much we are willing...
by Peter Ciszewski | May 3, 2019
Ilan Ganot, Co-Founder, President and CEO of Solid Biosciences provides an overview of his company and it’s focus on Duchenne muscular dystrophy (DMD). Mr. Ganot started Solid in 2013 to find treatments, and potentially a cure, for DMD, a disease that...
by Peter Ciszewski | May 3, 2019
Heather A. Lau, MD, Assistant Professor, Department of Neurology; Associate Director, Division of Neurogenetics; Director, Lysosomal Storage Disease Program at NYU Langone Health provides an overview of Mucopolysaccharidosis type II (MPS II), also known as...
