Neurometabolic Diseases Lab

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Neurometabolic Diseases Lab

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...
 

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 Sunday, 04 October 2020 11:24 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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