Metachromatic Leukodystrophy News and Research

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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

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 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

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

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

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

Rochester receives $1.2 million to battle rare childhood disease

Rochester receives $1.2 million to battle rare childhood disease

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