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March 07, 2022

Advancing Novel Treatments for Rare Neurological Diseases

In honor of Rare Disease Day 2022, The Oxford-Harrington Rare Disease Centre (OHC) hosted a Rare Disease Day Webinar, Advancing Novel Treatments for Neurological Rare Diseases, on Monday, February 28. The Oxford-Harrington Rare Disease Centre is a partnership between Harrington Discovery Institute at University Hospitals and the University of Oxford in London, England. This collaboration was formed out of the need to advance critical medicines for the more than 7,000 rare diseases without treatments or cures.

The webinar featured a panel of experts discussing areas of unmet needs in Neurological Rare Diseases, Rare Cancers, and Rare Developmental Disorders.

Richard Wade-Martins, MA, DPhil, Professor of Molecular Neuroscience, University of Oxford. Dr. Wade-Martins spoke about his research and target discovery related to Friedreich's ataxia, an inherited rare disorder that affects some of the body's nerves.

Haiyan Zhou, MD, PhD, Harrington Scholar, and Siew Ho, PhD, Harrington Advisor. Dr. Zhou and Dr. Ho discussed Harrington Discovery Institute's unique approach that brings in critical funding to drug discovery, along with expert guidance and mentorship in the drug development process from advisors on Harrington’s Therapeutics Development Center (TDC). Dr. Zhou’s work focuses on a therapy with the potential to cure (HSN1), a debilitating disorder of peripheral nerve that results in sensory loss, neuropathic pain, varying degrees of limb weakness, and mutilating skin ulceration.

Jonathan S. Stamler, MD, President, Harrington Discovery Institute, Robert S. and Sylvia K. Reitman Family Foundation Distinguished Professor of Cardiovascular Innovation and Professor of Medicine and of Biochemistry at University Hospitals and Case Western Reserve University, spoke about additional Harrington Scholars advancing treatments in Rare Disease supported by Harrington Discovery Institute. These scholars are focused on impacting the standard of care related to ALS (Amyotrophic Lateral Sclerosis), a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, Rett Syndrome, a rare genetic mutation affecting brain development in girls, and other rare diseases without cures.

Mr. Rusty Cooper and his family shared their personal experience of how they support Rusty's grandson who has an ultra-rare neurological disease and how advancements from Harrington Discovery Institute have given them hope.

To learn more and to become involved in our work creating cures, contact Tyrone Gorden, Vice President of Global Development, at Tyrone.Gorden@HarringtonDiscovery.org.