by Peter Ciszewski | Oct 15, 2019
Neena Nizar, PhD, founder of the Jansen’s Foundation, is the personification of the determination. Born with a rare disease that was not properly diagnosed until after her sons’ were also diagnosed with the condition (Jansen’s metaphyseal...
by Peter Ciszewski | Oct 14, 2019
Bobby Gasper, MD Chief Scientific Officer of Orchard Therapeutics, discusses his company’s gene therapy being developed for MPS I. Dr. Gasper noted while ERT is available for these children, that treatment does not cross the blood brain barrier and there is a...
by Peter Ciszewski | Oct 11, 2019
Patricia Welton has two daughters with Ehlers-Danlos syndrome. Her daughters had the disease for years before properly being diagnosed. That delay in diagnosis as well as the concept that each rare disease patient is unique and has a story to tell was the...
by Peter Ciszewski | Oct 10, 2019
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. Huntington...
by Peter Ciszewski | Oct 9, 2019
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. The grants, part of the Orphan Products Clinical Trials Grants...
