ResearchFocus on the pharmaceutical and biotech industry
Kristin Smedley is the President of Curing Retinal Blindness Foundation, a non-profit organization focused on finding treatments and cures for CRB1 retinal disease.
Joe Pugliese of the Hemophilia Alliance, a not-for-profit organization of federally funded hemophilia treatment centers that is working to ensure its member have the expertise, resources and public support to sustain their integrated clinical and pharmacy services for persons with hemophilia and other bleeding disorders.
Accelerating the Road to Treatment: The Global Commission to End the Diagnostic Odyssey for Children
The Global Commission to End the Diagnostic Odyssey for Children, co-chaired by Takeda, Microsoft and EURORDIS-Rare Diseases Europe, is a multidisciplinary group of patient advocates, physicians and other experts working together to help solve the complex challenges impacting the rare disease community.
Bobby Gasper, MD Chief Scientific Officer of Orchard Therapeutics, discussuses his company’s gene therapy being developed for MPS I.
Scott Schobel, MD, MSc, clinical science leader for Roche’s Huntington Disease Program talks about the role that Huntington disease patient group, throughout the world, helped Roche develop their clinical program focused on Huntington disease.
The U.S. Food and Drug Administration (FDA) announced it has awarded over $15 million in new clinical trial research grants to enhance the development of medical products for patients with rare diseases.
David Anderson, PhD, CNS Research Lead at Spark Therapeutics talks about the numerous neurological conditions that have been identified and the concern that most of them are rare diseases and most of them are not being properly managed.
Mark Rothera, CEO of Orchard Therapeutics, discusses ex vivo autologous hematopoietic stem cell gene therapies for adenosine deaminase severe combined immunodeficiency (ADA-SCID) and metachromatic leukodystrophy (MLD).
At Ovid Therapeutics, the patients’ input is essential to how they develop medications and how they do business. Leading that mindset is Luke Rosen, Patient Engagement & Digital Health Experience Head for the company.
Scott Schobel, MD, MSc, clinical science leader for Roche’s Huntington Disease Program, discusses RG6042, a RNA antisense drug that can reduce the synthesis of the Huntington protein and thereby, reduce its toxic effects in the brain.