Enzyme replacement therapy for alpha-mannosidosis: 12 months follow-up of a single centre, randomised, multiple dose study (Q39451308)

12 September 2017

Enzyme replacement therapy for alpha-mannosidosis: 12 months follow-up of a single centre, randomised, multiple dose study (English)

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enzyme replacement therapy

12 September 2017

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L Borgwardt

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12 September 2017

C I Dali

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12 September 2017

J Fogh

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12 September 2017

J E Månsson

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12 September 2017

K J Olsen

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12 September 2017

H C Beck

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12 September 2017

K G Nielsen

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12 September 2017

L H Nielsen

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12 September 2017

S O E Olsen

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12 September 2017

H M F Riise Stensland

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O Nilssen

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12 September 2017

F Wibrand

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12 September 2017

A M Thuesen

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T Pearl

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U Haugsted

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P Saftig

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J Blanz

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S A Jones

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12 September 2017

A Tylki-Szymanska

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N Guffon-Fouiloux

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12 September 2017

M Beck

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A M Lund

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12 September 2017

14 March 2013

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Journal of Inherited Metabolic Disease

12 September 2017

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12 September 2017

36

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12 September 2017

1015-1024

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12 September 2017

6

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https://scigraph.springernature.com/pub.10.1007/s10545-013-9595-1

12 September 2017

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https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

A step in the right direction: assessing exercise tolerance in cystic fibrosis.

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

An official American Thoracic Society/European Respiratory Society statement: pulmonary function testing in preschool children

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Reversal of peripheral and central neural storage and ataxia after recombinant enzyme replacement therapy in alpha-mannosidosis mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Tau protein in cerebrospinal fluid: a biochemical marker for axonal degeneration in Alzheimer disease?

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Nonverbal cognitive abilities and auditory performance in children fitted with auditory brainstem implants: preliminary report.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Review of the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2)

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

A phase 1/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studies

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Chemically modified beta-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

A solution to limitations of cognitive testing in children with intellectual disabilities: the case of fragile X syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Exercise testing, 6-min walk, and stair climb in the evaluation of patients at high risk for pulmonary resection

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Psychiatric symptoms in alpha-mannosidosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Converging molecular pathways in human neural development and degeneration.

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Prevalence of lysosomal storage disorders

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Late effects of early growth hormone treatment in Down syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

alpha-Mannosidosis: functional cloning of the lysosomal alpha-mannosidase cDNA and identification of a mutation in two affected siblings.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Safety and pharmacokinetics of agalsidase alfa in patients with Fabry disease and end-stage renal disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Efficacy of enzyme replacement therapy in alpha-mannosidosis mice: a preclinical animal study

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

A sensitive ELISA for glial fibrillary acidic protein: application in CSF of children.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Patients with Amyotrophic Lateral Sclerosis and Other Neurodegenerative Diseases Have Increased Levels of Neurofilament Protein in CSF

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Concurrent validity and classification accuracy of the Leiter and Leiter-R in low-functioning children with autism

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Review of three tests of motor proficiency in children

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Tau protein isoforms, phosphorylation and role in neurodegenerative disorders11These authors contributed equally to this work

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-013-9595-1

Review of the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2)

21 January 2018

1 reference

12 December 2020

Neurofilament light as a prognostic marker in multiple sclerosis

1 reference

12 December 2020

Alpha-mannosidosis in the guinea pig: a new animal model for lysosomal storage disorders

1 reference

12 December 2020

Developmental analysis of CNS pathology in the lysosomal storage disease alpha-mannosidosis

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12 December 2020

Patients with amyotrophic lateral sclerosis and other neurodegenerative diseases have increased levels of neurofilament protein in CSF.

1 reference

12 December 2020

Longitudinal cerebrospinal fluid biomarkers over four years in mild cognitive impairment

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12 December 2020

Stair climbing as an indicator of pulmonary function

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12 December 2020

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12 December 2020

Laronidase (Aldurazyme): enzyme replacement therapy for mucopolysaccharidosis type I

1 reference

12 December 2020

After the storm: neurofilament levels as a surrogate endpoint for neuroaxonal damage

1 reference

12 December 2020

10.1007/S10545-013-9595-1

Identifiers

DOI

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12 September 2017

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