Neurometabolic Diseases Lab

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Neurometabolic Diseases Lab

Un cóctel de antioxidantes, potencial terapia para la adrenoleucodistrofia

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From
February 3, 2011

Un grupo dirigido por Aurora Pujol, del Instituto de Investigación Biomédica de Bellvitge (Idibell), ha descubierto que la combinación de tres antioxidantes revierte los principales síntomas de la adrenoleucodistrofia ligada al cromosoma X. El ensayo piloto en humanos está en fase de reclutamiento y pendiente de aprobación para comenzar.

Last Updated on Friday, 11 February 2011 12:08 Read more...
 

Gene Therapy Halts Fatal Brain Disease

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From Wired Science
November 5, 2009
By Brandon Keim

Scientists have used gene therapy to halt the progression of adrenoleukodystrophy, a fatal neurodegenerative disease caused by a single defective gene, in two seven-year-old boys.

It took more than a decade to refine the therapy, in which stem cells taken from the boys’ bone marrow were hacked with healthy copies of the gene, then returned to their bodies. Without them, the boys would soon be dead.

Last Updated on Tuesday, 10 November 2009 12:02 Read more...
 

El ácido valproico es efectivo en adrenoleucodistrofia ligada al X

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From
March 12, 2010

Un estudio realizado en el Instituto de Investigación Biomédica de Bellvitge (Idibell) ha demostrado que el ácido valproico, actualmente indicado para tratar la epilepsia, es capaz de corregir los biomarcadores de daño oxidativo en pacientes con adrenoleucodistrofia ligada al X.

Last Updated on Friday, 11 February 2011 14:48 Read more...
 

Gene therapy could cure Lorenzo’s Oil brain disease

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From
November 6, 2009

A devastating brain disease that kills boys by the time of adolescence has been stopped in its tracks by a gene therapy that raises hope for thousands of families.

The treatment has halted the development of adrenoleukodystrophy (ALD) — the genetic disorder featured in the film Lorenzo’s Oil — in two Spanish boys aged 7 who would otherwise have suffered progressive and ultimately fatal brain damage. A third boy has also been treated, but too recently for doctors to know whether it has been successful.

Last Updated on Tuesday, 10 November 2009 11:56 Read more...
 


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Funding

Instituto de Salud Carlos III
CIBERER

ICREA
Myelinet
COST
Leukotreat
Olivers Army
The Myelin Project
Walk On Project

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