Tagged as: Leukodystrophy

bluebird bio to Present Clinical Data from Study of Lenti-D in ALD at CNS Annual Meeting

CAMBRIDGE, Mass.–(BUSINESS WIRE)–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 the initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™ investigational

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September is Leukodystrophy Awareness Month

September is Leukodystrophy Awareness Month! Share your story on Facebook, Twitter, and Instagram using the hashtag #LeukodystrophyAwareness and help us spread the word to ensure that leukodystrophies become a household name. Leukodystrophies are a group of rare, progressive, metabolic, genetic diseases that affect the brain, spinal cord and often the peripheral nerves.

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Researchers receive $1.9 million NIH grant to better understand leukodystrophies

A team of Wayne State University researchers recently received a $1.9 million grant from the National Eye Institute of the National Institutes of Health to better understand leukodystrophies (LD) and genetic Leukoencephalopathies (gLE), rare genetic disorders affecting the white matter — myelin — in the central nervous system. Patients diagnosed

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California A.L.D. Newborn Screening Program to Begin mid-September

We received word from the California Department of Public Health’s Genetic Disease Screening Program (GDSP) that adrenoleukodystrophy (ALD) screening will begin in mid-September. In 2013, The Myelin Project sponsored California Assembly Bill 1559 – authored by former Assemblyman, now California Senator Dr. Richard Pan – in order to mandate newborn screening of adrenoleukodystrophy (ALD) in California. On September 25th, 2014, Governor Jerry

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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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