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Leukodystrophies

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The family handed THREE death sentences: Parents are told their twin boys have the same genetic brain disease that’s slowly killing their daughter – Daily Mail

A couple have been left utterly heartbroken after all three of their children were diagnosed with a rare and deadly brain condition they…

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One seizure left her crippled – but doctors have no answers for girl, 10, as she battles incredibly rare brain disease that has no cure – Daily Mail

Jayda Suttie was in gym class when she collided heads with a boy while running. What would have been a bump on the head…

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Grant award earmarked for gene, blindness study – Life Science Daily

Wayne State University researchers recently received a $1.9 million grant as a means of gaining greater insight into leukodystrophies (LD) and genetic Leukoencephalopathies…

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Scientists Use Stem Cells to Grow Brain and Muscle Cells Faster Than Ever – Inverse

Stem cells are special. Present in abundant quantities during fetal development — and in smaller but still significant numbers in some adult organs…

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Boy, 7, with life limiting condition given the gift of a new garden – Lincolnshire Echo

A little boy who has a life-limiting condition will be…

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Experimental Biology 2016 Programming at a Glance – Newswise (press release)

However, recent research developments have brought hope that new drug or gene therapies for CNS diseases such as Down syndrome, autism, Alzheimer’s disease…

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Health mystery: A child’s odd teeth – gulfnews.com

Leukodystrophies affect the myelin sheath that insulates nerve cells, which are essential to the functioning of the brain and central nervous system. Myelin…

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Lorenzo’s Oil Could Not Cure Lorenzo, But Newborn Screening Is Expected To Save Others From His Fate – Forbes

In 2013, Kennedy Krieger opened the Moser Center for Leukodystrophies in his honor. “It was my late husband who saw the need for…

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Targeted gene addition in human CD34 + hematopoietic cells for correction of X-linked chronic granulomatous disease – Nature.com

Ex vivo transfer of therapeutic transgenes into autologous HSPCs using viral vectors has benefitted patients with rare genetic conditions, such as the leukodystrophies…

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Hope, Hype, and Wishful Thinking – University of Rochester Newsroom (blog)

Diseases of the white matter – like MS and other rare conditions like pediatric leukodystrophies and white matter stroke – are considered strong…

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A mother’s internet sleuthing about her daughter’s odd teeth leads to a troubling discovery – Sydney Morning Herald

McClintock “basically told us that we may never know exactly what is going on,” Rennon recalled; doctors did not know what kind of…

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A mum’s discovery about her daughter’s odd teeth leads to surprising diagnosis – Stuff.co.nz

Her case was transferred to Vanderver, an international expert in leukodystrophies who directs the programme. For Erica’s parents, the news was devastating. “It…

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Leukodystrophies – Pipeline Review, Market Growth, Segment, Trends and Forecast 2016 – 2020 – Medgadget.com (blog)

MRS275 “Leukodystrophies Global 2015 Clinical Trials Review, H2” provides an detailed overview of Leukodystrophies scenario. Report includes top line data relating on Leukodystrophies…

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MUHC says it made breakthrough to treat degenerative disease – Globalnews.ca

WATCH: MUHC researchers say they made a major breakthrough towards curing a rare and incurable degenerative illness. Global’s Billy Shields reports. MONTREAL –…

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The Children’s Hospital of Philadelphia to Launch Leukodystrophy Center of Excellence for Children with Degenerative … – PR Newswire (press release)

PHILADELPHIA, May 19, 2015 /PRNewswire-USNewswire/ — The Children’s Hospital of Philadelphia (CHOP) announced the launch of a Leukodystrophy Center of Excellence at the…

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Children’s Hospital of Pittsburgh Foundation Receives $2.5 Million Grant from The Legacy of Angels Foundation to … – PR Newswire (press release)

Started in 2000, the program focuses on genetic neurodegenerative diseases, such as Krabbe disease, an inherited condition caused by a faulty gene, and…

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A mum’s discovery about her daughter’s odd teeth leads to a troubling discovery – Stuff.co.nz

Her case was transferred to Vanderver, an international expert in leukodystrophies who directs the programme. For Erica’s parents, the news was devastating. “It…

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Odd teeth: A mother’s Internet sleuthing led to her daughter’s troubling diagnosis – Washington Post

Leukodystrophies affect the myelin sheath that insulates nerve cells, which are essential to the functioning of the brain and central nervous system. Myelin…

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Research and Markets: Leukodystrophies Therapeutics Pipeline Review 2015 – 6 Companies & 6 Drug Profiles – Business Wire (press release)

This report provides comprehensive information on the therapeutic development for Leukodystrophies, complete with comparative analysis at various stages, therapeutics assessment by drug target,…

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Wendy’s windows used to help Hunter’s Hope – Lockport Union-Sun & Journal

North Park eighth-grader Ellie Igoe, 13, paints hearts on one of Wendy’s windows. North Park art students paint the South Transit Road restaurant’s…

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