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Making Miracles Possible for Kids with Leukodystrophy

In 2013, a year after Pat and Maria Carr learned that their youngest child had one of the 50 forms of leukodystrophy, a degenerative, genetic neurological condition, they knew they wanted to help.  It was too late to save Cal, but the Carrs were determined to give children with leukodystrophy

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bluebird bio Publishes Interim Data from Starbeam Study of Lenti-DTM Drug in Cerebral Adrenoleukodystrophy (CALD) Patients

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Oct. 4, 2017– bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, today announced that interim data from an initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™

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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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