Caroline has a rare and severe form of Charcot-Marie-Tooth (CMT) disease, a neurodegenerative disorder that is deteriorating her muscles.
By the age of three, Talia was still not able to walk on her own. Years later, she was finally diagnosed with CMT4J, a very rare form of Charcot-Marie-Tooth disease.
Caroline, age 7, and Talia, age 13, each has separate, very rare forms of Charcot-Marie-Tooth disease, a neurodegenerative disorder. There’s no known cure for either form of the disease – yet.
JAX scientists took on the challenge of creating a cure for Caroline, and one for Talia, testing a novel gene therapy on mouse models made specifically to mimic each girl’s disease. The mice improved, showing the promise of new gene therapy. Both girls’ families are now seeking FDA approval for a clinical trial.
Our approach, while tailored to patients like Caroline and Talia, may also be applied to other diseases, and holds lifesaving potential for both girls – and others.
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