Early treatment is associated with improved cognition in Hurler syndrome. (Q50354624)

6 March 2018

title

Early treatment is associated with improved cognition in Hurler syndrome. (English)

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6 March 2018

author name string

Michele D Poe

series ordinal

1

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6 March 2018

Sarah L Chagnon

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2

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Maria L Escolar

series ordinal

3

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6 March 2018

publication date

8 October 2014

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6 March 2018

published in

Annals of Neurology

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6 March 2018

volume

76

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page(s)

747-753

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issue

5

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The clinical outcome of Hurler syndrome after stem cell transplantation.

cites work

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21 January 2018

1 reference

Hurler disease (mucopolysaccharidosis type IH): clinical features and consanguinity in Tunisian population

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Molecular genetics of mucopolysaccharidosis type I: mutation analysis among the patients of the former Soviet Union

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1 reference

Mucopolysaccharidosis type I in 21 Czech and Slovak patients: mutation analysis suggests a functional importance of C-terminus of the IDUA protein.

21 January 2018

1 reference

IDUA mutational profiling of a cohort of 102 European patients with mucopolysaccharidosis type I: identification and characterization of 35 novel α-L-iduronidase (IDUA) alleles.

21 January 2018

1 reference

Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation

21 January 2018

1 reference

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1 reference

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Outcome in six children with mucopolysaccharidosis type IH, Hurler syndrome, after haematopoietic stem cell transplantation (HSCT).

21 January 2018

1 reference

Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.

21 January 2018

1 reference

Pre-transplant risk factors affecting outcome in Hurler syndrome.

21 January 2018

1 reference

Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome

21 January 2018

1 reference

Oral busulfan pharmacokinetics and engraftment in children with Hurler syndrome and other inherited metabolic storage diseases undergoing hematopoietic cell transplantation

21 January 2018

1 reference

Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources

21 January 2018

1 reference

Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres

21 January 2018

1 reference

Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation

21 January 2018

1 reference

Long-term outcome of Hurler syndrome following bone marrow transplantation.

21 January 2018

1 reference

An exploratory study of brain function and structure in mucopolysaccharidosis type I: long term observations following hematopoietic cell transplantation (HCT)

21 January 2018

1 reference

Variable disease progression after successful stem cell transplantation: prospective follow-up investigations in eight patients with Hurler syndrome.

21 January 2018

1 reference

Cognitive and psychosocial functioning of pediatric hematopoietic stem cell transplant patients: a prospective longitudinal study.

21 January 2018

1 reference

Cognitive and academic consequences of stem-cell transplantation in children.

21 January 2018

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21 January 2018

Identifiers

DOI

10.1002/ANA.24246

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6 March 2018

PubMed ID

25103575

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6 March 2018