by Peter Ciszewski | Jan 8, 2020
Gallia Levy, MD, PhD, Associate Group Medical Director, at Genentech explains why joint pain is so prevalent in persons with Hemophilia A. Hemophilia A is a genetic disorder caused by missing or defective clotting protein, factor VIII. Symptoms are dependent on...
by Peter Ciszewski | Jan 7, 2020
Rajiv Ratan, MD, PhD, of the Burke Neurological Institute, explains the mechanisms involved in how cells undergo ‘programmed death’ following a hemorrhagic stroke. A hemorrhagic stroke occurs when a blood vessel bursts and blood leaks into the surrounding brain...
by Peter Ciszewski | Jan 6, 2020
Gaurav Shah, MD, Chief Executive Officer & President of Rocket Pharmaceuticals explains the advantages that smaller biotech companies have in developing treatments for rare diseases. Rocket Pharmaceuticals is currently developing gene therapies for a number...
by Peter Ciszewski | Jan 3, 2020
Brigid Brennan of the Friedreich’s Ataxia Research Alliance (FARA) discusses the origins of this patient organization and its mission to focus on research about Friedreich’s ataxia. Some of the research FARA is funding includes a natural history study to better...
by Peter Ciszewski | Jan 2, 2020
David Pearce, PhD, of the Coordination of Rare Diseases at Sanford (CoRDS) talks about Sanford Health, one of the largest health systems in the United States. Sanford Health provides access to integrated health care, genomic medicine, senior care and services,...