by Peter Ciszewski | Jun 12, 2019
Ilan Ganot, Co-Founder, President and CEO of Solid Biosciences discusses how his company views advocates and patients who have 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 | Jun 11, 2019
Kristen Wheeden, Director of Development for the American Porphyria Foundation, provides an overview of her foundation and it’s focus on physician education. The American Porphyria Foundation is also dedicated to the health and well-being of patient and...
by Peter Ciszewski | Jun 10, 2019
Harrison Brown, PhD, BBA, is the Chief Science Officer at Odylia Therapeutics, a non-profit corporation working to help develop gene therapies for rare eye disease. As Dr. Brown explains in this video, gene therapy is ideal suited for many rare diseases since...
by Peter Ciszewski | Jun 10, 2019
Mark Pykett, PhD, Chief Scientific Officer at PTC Therapeutics, provides an overview of his company, including its focus on rare diseases. Mark leads the research initiatives of PTC. Before joining the company in August 2018, Mark held a number of executive...
by Peter Ciszewski | Jun 8, 2019
Malissa J. Wood, MD is co-director of the Corrigan Women’s Heart Health Program at the Massachusetts General Hospital Heart Center and associate professor of medicine at Harvard Medical School. In this video, Dr. Wood discusses the iSCAD Registry, which...
