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Small molecule helps fix ‘Lorenzo’s Oil’ gene mutation

Sobetirome partially makes up for lost protein function By: Stu Borman Researchers have identified a small organic molecule that may help fix the genetic defect that causes X-linked adrenoleukodystrophy (X-ALD), the rare disease featured in the 1992 movie “Lorenzo’s Oil.” X-ALD, which affects 1 in 20,000 to 50,000 people worldwide, typically

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The Leukodystrophy Care Network (LCN) – Establishing Care Guidelines

Imagine your child experiencing an unexplained sudden loss of basic functions – a toddler who once constantly walked and ran suddenly stumbling with each step; an infant with a healthy appetite who once smiled and cooed now crying inconsolably for every waking hour and only eating a few ounces of

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