Defect in debilitating neurodegenerative disease reversed in mouse nerves

Gerald Dorn, PhD, left, and staff scientist Agostinho Rocha, study mitochondria in relation to Charcot Marie Tooth disease in Dorn's lab in the McDonnell Medical Sciences Building on March 22, 2018. MILLER/WASHINGTON UNIVERSITY SCHOOL OF MEDICINE

Source: the Source – April 19, 2018

Scientists have developed a new drug compound that shows promise as a future treatment for Charcot-Marie-Tooth disease, an inherited, often painful neurodegenerative condition that affects nerves in the hands, arms, feet and legs. The researchers used the compound to treat the nerves of mice harboring the genetic defects that cause the disease.

The new study, from Washington University School of Medicine in St. Louis, challenges some conventional wisdom regarding how patients with this disease lose the ability to move their limbs.

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