Could Statins Ease Deadly Heart Condition in Rare Neuromuscular Disease?

PHILADELPHIA – In preclinical studies using cell models that mimicked liver cells of patients with the rare disease Friedreich’s ataxia (FA), a widely used cholesterol-lowering drug increased a precursor of HDL (high-density lipoprotein), the “good cholesterol,” according to new research published in PLOS One from the Perelman School of Medicine at the University of Pennsylvania.

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Amicus Establishes Global Research and Gene Therapy Center of Excellence in Philadelphia

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James Wilson, MD, PhD, from the University of Pennsylvania, Joins the Rett Syndrome Research Trust’s Gene Therapy Consortium