Tagged as: gene therapy

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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The Myelin Project & Cure ALD Foundation Grant $50,000 to Dr. Florian Eichler’s AMN Gene Therapy Study

The Myelin Project in partnership with the Cure ALD Foundation, has granted $50,000 to Dr. Florian Eichler’s pilot study of gene therapy in adrenomyeloneuropathy (AMN). Dr. Eichler and his team have established an AMN animal model proof of concept showing a gene therapy approach could provide benefit where no treatment is currently

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Bluebird Bio’s Gene Therapy Shows Promise for ALD Treatment

By: Ben Fidler April 20th, 2016 BOSTON – Cerebral adrenoleukodystrophy is a rare, crippling neurological disease that leaves its patients, typically boys, severely disabled and ultimately dead in a matter of years. Only painful, dangerous bone marrow transplants can stop the progression of CALD. But while the transplants are effective

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