Leukodystrophy News and Research

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Leukodystrophies are inherited disorders that are characterized by a progressive breakdown (demyelination or hypomyelination) of the white matter in the central nervous system, caused by different mechanisms involving myelin proteins, as well as lipid and organic acid metabolism. They have to be distinguished from the “umbrella” term leukoencephalopathy, which is used to delineate any disease of the white matter.

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Researchers develop new method to capture how brain cells interact

Researchers develop new method to capture how brain cells interact

International research team identifies new gene associated with 4H leukodystrophy

International research team identifies new gene associated with 4H leukodystrophy

UC Davis researchers settle long-standing controversy surrounding Canavan disease

UC Davis researchers settle long-standing controversy surrounding Canavan disease

FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

State highlights: Calif.'s Prop. 45 fight; new benefits for same sex couples in N.C.; Aetna sheds jobs after Medicaid drop

State highlights: Calif.'s Prop. 45 fight; new benefits for same sex couples in N.C.; Aetna sheds jobs after Medicaid drop

Cancer drug shows promise in treating blood cell disorders

Cancer drug shows promise in treating blood cell disorders

Researchers develop adjuvant which enhances effect of virus transduction

Researchers develop adjuvant which enhances effect of virus transduction

Manchester scientists treat fatal genetic brain disease using stem cell gene therapy

Manchester scientists treat fatal genetic brain disease using stem cell gene therapy

'Defanged' HIV used to deliver gene therapy

'Defanged' HIV used to deliver gene therapy

First Edition: July 12, 2013

First Edition: July 12, 2013

Drug used to control Type II diabetes can help repair spinal cords of mice suffering from inherited disease

Drug used to control Type II diabetes can help repair spinal cords of mice suffering from inherited disease

IDIBELL signs patent licensing agreement with Minoryx

IDIBELL signs patent licensing agreement with Minoryx

New treatment strategy for patients with CMT disease on the horizon

New treatment strategy for patients with CMT disease on the horizon

Reprogrammed human skin cells highly effective in treating myelin disorders

Reprogrammed human skin cells highly effective in treating myelin disorders

Parents must be considered during expansion of genetic screening programs for newborns

Parents must be considered during expansion of genetic screening programs for newborns

Study identifies function of GlialCAM gene in brain edema

Study identifies function of GlialCAM gene in brain edema

Age-associated decline of remyelination process may be reversible

Age-associated decline of remyelination process may be reversible

Polystyrene surface triples number of human ES and iPS cells in culture

Polystyrene surface triples number of human ES and iPS cells in culture

Study provides blueprint for design of new HIV drugs

Study provides blueprint for design of new HIV drugs

ISWG researchers establish guidelines for diagnosis and treatment of infantile spasms

ISWG researchers establish guidelines for diagnosis and treatment of infantile spasms