Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure. (Q38787918)

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20 March 2020

title

Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure (English)

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9

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Simon Jones

10

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7

Roberto Giugliani

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20 March 2020

author name string

Johanna H van der Lee

1

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Jonathan Morton

2

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Heather R Adams

3

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Lorne Clarke

4

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Berendine Johanne Ebbink

5

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Maria L Escolar

6

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Paul Harmatz

8

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Shauna Kearney

11

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Joseph Muenzer

12

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Stewart Rust

13

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Margaret Semrud-Clikeman

14

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Frits A Wijburg

15

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Zi-Fan Yu

16

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Darren Janzen

17

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Elsa Shapiro

18

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publication date

6 May 2017

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Molecular Genetics and Metabolism

20 March 2020

published in

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volume

121

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page(s)

70-79

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issue

2

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Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.

20 March 2020

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10.1016/J.YMGME.2017.05.004

7 January 2021

Identifiers

DOI

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20 March 2020

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