Tagged as: Leukodystrophy

Can Two Brothers Struck With ‘Lorenzo’s Oil’ Disease Be Saved?

One brother is living a normal life; the other isn’t. Their story illustrates the potential of gene therapy. Source: The Daily Beast | By: KAREN WEINTRAUB | 11.01.17 9:00 AM ET In the early months of 2014, Brandon Rojas was a typical 6-year-old: healthy with a sunny disposition, a love of sports,

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ALD Mom & Board Member Janis Sherwood Featured on NPR’s “All Things Considered” for Newborn Screening Advocacy

To listen to the radio program, click here. The following article was written on www.npr.org by Anna Gorman: Kerri De Nies received the news this spring from her son’s pediatrician: Her chubby-cheeked toddler has a rare brain disorder. She’d never heard of the disease — adrenoleukodystrophy, or ALD — but soon felt

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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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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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