2023 Global Rare Disease Day Webinar
Advancing Novel Treatments for Rare Diseases
Individuals with rare diseases and their families typically have a long journey to a diagnosis with little hope for a cure. The Oxford-Harrington Rare Disease Centre, a partnership between the University of Oxford and Harrington Discovery Institute, brings together the resources and capabilities needed to accelerate novel treatments for patients living with a rare disease.
A special rare disease day webinar was hosted on Monday, February 27, 2023 in honor of Rare Disease Day to highlight the OHC’s mission and progress towards advancing cures for rare diseases.
Individually Rare, Collectively Common
350 million people across the globe have a rare disease, affecting 1 in 17 people during their lifetime. Although individual rare diseases are rare, collectively they represent about one third of human disease. Fewer than 5% of rare diseases have approved treatments.
The OHC supports the drug development life cycle from early drug discovery to preclinical studies, clinical development, regulatory approval and ultimately through to commercialization.
“This affiliation represents a commitment to patients first and a tremendous opportunity to improve the health and outcomes of those living with a rare disease,” said Jonathan S. Stamler, MD, President, Harrington Discovery Institute.
Turning Potential into Impact
Rare diseases collectively represent about one third of human disease. Approximately 400 million people worldwide suffer from a rare disease, and the majority are children. The OHC is accelerating the development of new treatments through a major transatlantic initiative.
“There are big challenges in meeting the needs of rare diseases. With advances in genomics and drug development, we are now in a position to make a difference for people who have had little hope of finding a treatment,” says Professor Matthew Wood, Director of the OHC.
Rare disease research and drug discovery requires collaboration between patients, organizations, researchers and clinicians. Only then can treatments for the millions of people with a rare disease become a reality. The webinar included voices from all of these collective advocates, including:
Angela Russell, DPhil, Harrington Scholar and William Greenlee, PhD, Harrington Advisor. Professor Russell and Dr. Greenlee discussed Harrington Discovery Institute’s unique approach that brings in funding for drug discovery, along with expert guidance and mentorship from advisors in Harrington’s Therapeutics Development Center (TDC). Professor Russell’s work is about advancing a novel treatment for Duchenne Muscular dystrophy (DMD).
Julia Vitarello, Founder and CEO, Mila’s Miracle Foundation. Ms. Vitarello spoke of her experience as a family advocate for rare disease treatments, with a specific focus on Batten disease.
Jonathan Stamler, MD, President and Founder of Harrington Discovery Institute. Dr. Stamler shared updates about the vision of The OHC in its global quest to advance cures for rare diseases.
Matthew Wood, Professor, University of Oxford and Director, Oxford-Harrington Rare Disease Centre. Professor Wood spoke of OHC's mission, vision and recent accomplishments.
Diana Wetmore, PhD, Vice President of Harrington Discovery Institute's Therapeutics Development Center. Dr. Wetmore summarized Harrington's experience with advancing rare disease treatments.