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

Shire's clinical study results for treatment of Gaucher disease to be presented at LDN World Symposium

Shire's clinical study results for treatment of Gaucher disease to be presented at LDN World Symposium

Daval International receives award for its anti-inflammatory agent AIMSPRO

Daval International receives award for its anti-inflammatory agent AIMSPRO

Gene therapy saves two children with adrenoleukodystrophy

Gene therapy saves two children with adrenoleukodystrophy

New technique to treat metabolic disorders in babies while still in the womb

New technique to treat metabolic disorders in babies while still in the womb

Research to explore diagnosis and treatment of rare diseases

Research to explore diagnosis and treatment of rare diseases

New hope for Batten disease sufferers

New hope for Batten disease sufferers

New ray of hope for children with Batten disease

New ray of hope for children with Batten disease

Umbilical cord blood transplant may confer advantages over bone marrow transplant for kids with life-threatening metabolic disorders

Umbilical cord blood transplant may confer advantages over bone marrow transplant for kids with life-threatening metabolic disorders

Rochester receives $1.2 million to battle rare childhood disease

Rochester receives $1.2 million to battle rare childhood disease

New hope for Krabbe disease sufferers

New hope for Krabbe disease sufferers

Cell therapy slows progression of metachromatic leukodystrophy

Cell therapy slows progression of metachromatic leukodystrophy

Infants with Krabbe disease saved by cord blood stem cells

Infants with Krabbe disease saved by cord blood stem cells