RARE Daily

Oxford-Harrington Rare Disease Centre to Advance Novel Treatment for Duchenne

August 3, 2022

The Oxford-Harrington Rare Disease Centre, a partnership of the University of Oxford and Harrington Discovery Institute at University Hospitals in Cleveland, Ohio, said it is supporting the development of a novel treatment for the rare neuromuscular disease Duchenne muscular dystrophy.

Photo: Angela Russell, professor of medicinal chemistry at the University of Oxford

Duchenne muscular dystrophy (DMD) primarily affects boys, usually beginning around the age of four. DMD is caused by a defect in a gene that makes dystrophin, which acts like a shock absorber when muscles contract. Without dystrophin, muscles progressively become damaged and weakened. Children with DMD experience difficulty standing up, walking, and may eventually require a wheelchair. Serious life-threatening complications may ultimately develop, including disease of the heart muscle and breathing difficulties. Patients with DMD typically do not live past their 30’s.

The research project is led by Angela Russell, professor of medicinal chemistry at the University of Oxford and a 2021 Harrington UK Rare Disease Scholar. She and her team have been working on a protein called utrophin, which was found to function similarly to dystrophin in protecting the muscle.

The lab has discovered new classes of molecules that increase utrophin production and has been working with Harrington’s Therapeutics Development Center since 2021 to advance the work. Russell’s project was recently selected by the Oxford-Harrington Rare Disease Centre for further drug development and commercialization support.

“Professor Russell’s project underscores our focus on new therapies that will prevent or reverse the debilitating effects of pediatric rare diseases,” said Matthew Wood, director of the Oxford-Harrington Rare Disease Centre.

With just 5 percent of rare diseases having an approved therapy to treat them, the Oxford-Harrington Rare Disease Centre was established to accelerate treatments in order to address the unmet need and improve the health and outcomes of patients living with a rare disease.

A project is considered for support by the Oxford-Harrington Rare Disease Centre if it offers a novel treatment for a rare disease, particularly those that have a genetic basis or affect children. Additional considerations include the potential for clinical impact and ability to impact other diseases. Through the center, successful principal investigators may qualify for additional funding to accelerate drug development, in addition to receiving drug and business development support.

Author: Rare Daily Staff

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