Tag: <span>Adrenoleukodystrophy</span>

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New drug candidate slows the progression of adrenoleukodystrophy
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New drug candidate slows the progression of adrenoleukodystrophy

by Paris Brain Institute Credit: Pixabay/CC0 Public Domain The work of Professor Fanny Mochel (AP-HP, Sorbonne University) at Paris Brain Institute, in collaboration with clinical research teams in eight countries and Spanish biotech Minoryx Therapeutics, has demonstrated the protective effects of leriglitazone in the progression of adrenoleukodystrophy—a rare genetic disease in which the white matter of the central...

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Stem cell gene therapy safe for adrenoleukodystrophy

(HealthDay)—Early results suggest that stem cell gene therapy is a safe and effective treatment for boys with early-stage cerebral adrenoleukodystrophy, according to a study published online Oct. 4 in the New England Journal of Medicine to coincide with the 2017 Child Neurology Society Annual Meeting, being held Oct. 4 to 7 in Kansas City. Florian Eichler, M.D.,...