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.

Further Reading

Researchers discover molecular underpinnings of Alexander disease

Researchers discover molecular underpinnings of Alexander disease

NIH grants boost rare diseases research efforts

NIH grants boost rare diseases research efforts

Scientists uncover the cause of a rare fatal nerve disease

Scientists uncover the cause of a rare fatal nerve disease

Scientists pinpoint cause of fatal genetic disorder in children

Scientists pinpoint cause of fatal genetic disorder in children

Novel palliative care approach for caregivers of children with rare diseases shows preliminary success

Novel palliative care approach for caregivers of children with rare diseases shows preliminary success

New, more efficient method for generating brain stem cells in the lab

New, more efficient method for generating brain stem cells in the lab

Salk research in yeast leads to serendipitous finding about hypomyelinating leukodystrophy

Salk research in yeast leads to serendipitous finding about hypomyelinating leukodystrophy

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

Wayne State researchers receive $1.9 million NIH grant to better understand rare genetic disorders

Wayne State researchers receive $1.9 million NIH grant to better understand rare genetic disorders

Specific gene pathway appears to regulate growth, structure, and organization of cerebral cortex

Specific gene pathway appears to regulate growth, structure, and organization of cerebral cortex

URMC researchers identify potential new means of treating severe genetic diseases in children

URMC researchers identify potential new means of treating severe genetic diseases in children

Cell therapy product can accelerate brain remyelination in mice

Cell therapy product can accelerate brain remyelination in mice

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

New tests could improve diagnosis of two rare childhood diseases

New tests could improve diagnosis of two rare childhood diseases

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