A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA. (Q38864028)

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title

A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA (English)

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1

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author name string

Catherine Breen

2

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Fiona Heap

3

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Stewart Rust

4

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Jessica de Ruijter

5

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Evelien Tump

6

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Jan Pieter Marchal

7

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Luying Pan

8

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Yongchang Qiu

9

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Jou-Ku Chung

10

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Nitin Nair

11

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Patrick A J Haslett

12

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Ann J Barbier

13

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Frits A Wijburg

14

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publication date

10 May 2016

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Molecular Genetics and Metabolism

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published in

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volume

118

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page(s)

198-205

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issue

3

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Cognitive development in patients with Mucopolysaccharidosis type III (Sanfilippo syndrome).

2 April 2020

cites work

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https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.006

Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations

7 January 2021

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Safety evaluation of chronic intrathecal administration of heparan N-sulfatase in juvenile cynomolgus monkeys.

7 January 2021

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A novel LC-MS/MS assay for heparan sulfate screening in the cerebrospinal fluid of mucopolysaccharidosis IIIA patients

7 January 2021

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Methods of neurodevelopmental assessment in children with neurodegenerative disease: Sanfilippo syndrome

7 January 2021

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A prospective study of catheter-related complications of intrathecal drug delivery systems

7 January 2021

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Programmable intrathecal opioid delivery systems for chronic noncancer pain: a systematic review of effectiveness and complications

7 January 2021

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Complications of intrathecal baclofen pumps in children

7 January 2021

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

7 January 2021

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Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, o

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.006

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.006

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2016.05.006

Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome).

7 January 2021

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10.1016/J.YMGME.2016.05.006

7 January 2021

Identifiers

DOI

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2 April 2020

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