Adrenoleukodystrophy News and Research

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Adrenoleukodystrophy (ALD) is one of a group of genetic disorders called the leukodystrophies that cause damage to the myelin sheath, an insulating membrane that surrounds nerve cells in the brain.

Genetix Pharmaceuticals develops a gene therapy product to treat ALD

Genetix Pharmaceuticals, a leader in gene therapy of somatic stem cells, announced today the company’s clinical program to treat Adrenoleukodystrophy (ALD). The company is developing a gene therapy product in collaboration with Professor Patrick Aubourg and Doctor Nathalie Cartier of the National Institute of Health and Medical Research (INSERM).

7 Nov 2009

Gene therapy saves two children with adrenoleukodystrophy

The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regaring the gene therapy in adrenoleukodystrophy conducted in France have just been published in the prestigious journal Science. Two children have been treated and their diseases have been halted. The children are doing well, which is unexpected for a disease destroying the brain in a few months. This discovery opens up treatment perspectives for numerous widespread diseases.

6 Nov 2009

Human stem cells show promise against fatal children's diseases

Scientists have used human stem cells to dramatically improve the condition of mice with a neurological condition similar to a set of diseases in children that are invariably fatal, according to an article in the June issue of the journal Cell Stem Cell.

4 Jun 2008

New approaches to genetic disease, based on cells' own ability to correct themselves

New approaches to genetic disease, based on cells' own ability to correct themselves, will be outlined today at the annual conference of the European Society of Human Genetics in Amsterdam, The Netherlands.

8 May 2006

Study proves Lorenzo's Oil prevents rare disease in boys

U.S. researchers have confirmed that a treatment called Lorenzo's Oil can prevent the onset of a rare but devastating neurological disease in young boys, X-linked adrenoleukodystrophy or X-ALD.

12 Jul 2005

Lorenzo's oil prevents onset of pediatric neurological disorder

Use of Lorenzo's Oil in young boys who have been diagnosed with but are not yet showing signs of a pediatric neurological disorder known as X-linked adrenoleukodystrophy (X-ALD) may prevent the disease from developing in the body. According to a study of male children published today in the Archives of Neurology, use of the oil prevented onset of the disease in a majority of cases.

11 Jul 2005

Infants with Krabbe disease saved by cord blood stem cells

Children with a fatal genetic disorder called Krabbe Disease can be saved and their brain development preserved if they receive stem cells from umbilical cord blood before symptoms of the disease develop, according to a study published in the current issue of the New England Journal of Medicine.

20 May 2005