Treatment of Rett Syndrome with Lovastatin

Aleksandra Djukic, MD, PhD Albert Einstein College of Medicine, Children’s Hospital at Montefiore

$403,000 AWARDED

Aleksandra Djukic, MD, PhD, medical director of the Tri-State Rett Syndrome Center at the Children’s Hospital at Montefiore is currently recruiting patients for a Phase 2 dose escalating open label clinical trial of lovastatin, a cholesterol lowering medication. The scientific basis for this trial stems from experiments conducted in the lab of mouse geneticist, Monica Justice, PhD, who identified the cholesterol pathway as a potential avenue to improve Rett symptoms.  The trial will determine the effect of lovastatin on gait, respiratory function, cognition, EEG and severity of the disease.

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