(Q34441882)

2 August 2017

Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial (English)

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2 August 2017

phase III clinical trial

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author

Vassili Valayannopoulos

6

object named as

Vassili Valayannopoulos

1 reference

2 August 2017

Eugen Mengel

4

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2 August 2017

2

Roberto Giugliani

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2 August 2017

Christian J Hendriksz

1

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2 August 2017

Paul Harmatz

3

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2 August 2017

Nathalie Guffon

5

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2 August 2017

Rossella Parini

7

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2 August 2017

Derralynn Hughes

8

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2 August 2017

Gregory M Pastores

9

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2 August 2017

Heather A Lau

10

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2 August 2017

Moeenaldeen D Al-Sayed

11

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2 August 2017

Julian Raiman

12

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2 August 2017

STRIVE Investigators

13

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2 August 2017

Ke Yang

14

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2 August 2017

Matthew Mealiffe

15

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2 August 2017

Christine Haller

16

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2 August 2017

6 September 2014

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Molecular Genetics and Metabolism

2 August 2017

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2 August 2017

114

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2 August 2017

178-185

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2 August 2017

2

1 reference

Incidence of the mucopolysaccharidoses in Northern Ireland

2 August 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Incidence of the mucopolysaccharidoses in Western Australia

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Molecular testing of 163 patients with Morquio A (Mucopolysaccharidosis IVA) identifies 39 novel GALNS mutations

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Subjective and Objective Assessment of Hand Function in Mucopolysaccharidosis IVa Patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Gait pattern and lower extremity alignment in children with Morquio syndrome

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

International Morquio A Registry: clinical manifestation and natural course of Morquio A disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Burden of disease in patients with Morquio A syndrome: results from an international patient-reported outcomes survey

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Transplant outcomes in mucopolysaccharidoses

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Outcomes of haematopoietic stem cell transplantation for inherited metabolic disorders: a report from the Australian and New Zealand Children's Haematology Oncology Group and the Australasian Bone Marrow Transplant Recipient Registry

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

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.2014.08.012

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Standardization of Spirometry, 1994 Update. American Thoracic Society

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Procedures for comparing samples with multiple endpoints

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Growth charts for patients affected with Morquio A disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age--a sibling control study

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

Effects of idursulfase enzyme replacement therapy for Mucopolysaccharidosis type II when started in early infancy: comparison in two siblings

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2014.08.012

10.1016/J.YMGME.2014.08.012

7 January 2021

Identifiers

DOI

1 reference

2 August 2017

1 reference