(Q37950844)

25 August 2017

The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus (English)

1 reference

enzyme replacement therapy

25 August 2017

0 references

mucopolysaccharidosis II

9 references

12 March 2019

14 March 2019

21 March 2019

24 March 2019

22 April 2019

25 April 2019

17 June 2019

25 June 2019

28 September 2019

6

Roberto Giugliani

1 reference

25 August 2017

Joseph Muenzer

1

1 reference

25 August 2017

Olaf Bodamer

2

1 reference

25 August 2017

Barbara Burton

3

1 reference

25 August 2017

Lorne Clarke

4

1 reference

25 August 2017

Gudrun Schulze Frenking

5

1 reference

25 August 2017

Simon Jones

7

1 reference

25 August 2017

Maria Verónica Muñoz Rojas

8

1 reference

25 August 2017

Maurizio Scarpa

9

1 reference

25 August 2017

Michael Beck

10

1 reference

25 August 2017

Paul Harmatz

11

1 reference

25 August 2017

29 October 2011

1 reference

European Journal of Pediatrics

25 August 2017

1 January 2012

9 references

12 March 2019

14 March 2019

21 March 2019

24 March 2019

22 April 2019

25 April 2019

17 June 2019

25 June 2019

28 September 2019

10 references

25 August 2017

12 March 2019

14 March 2019

21 March 2019

24 March 2019

22 April 2019

25 April 2019

17 June 2019

25 June 2019

28 September 2019

171

1 reference

25 August 2017

1

1 reference

25 August 2017

181-188

1 reference

https://scigraph.springernature.com/pub.10.1007/s00431-011-1606-3

25 August 2017

0 references

cites work

Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Transplant outcomes in mucopolysaccharidoses

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Multidisciplinary management of Hunter syndrome.

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Mucopolysaccharidosis I: management and treatment guidelines

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome).

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

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

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1.

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Molecular basis of iduronate-2-sulphatase gene mutations in patients with mucopolysaccharidosis type II (Hunter syndrome).

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Mutation analysis in 57 unrelated patients with MPS II (Hunter's disease)

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

A clinical and genetic study of Hunter's syndrome. 2. Differences between the mild and severe forms.

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Mild form of Hunter's syndrome: clinical delineation based on 31 cases

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Hunter syndrome presenting as macrocephaly and hydrocephalus

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

6 September 2017

Mucopolysaccharidoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase).

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Identification of iduronate sulfatase gene alterations in 70 unrelated Hunter patients.

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Mutation analysis in the iduronate-2-sulphatase gene in 43 Japanese patients with mucopolysaccharidosis type II (Hunter disease).

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Bone marrow transplantation in Hunter syndrome.

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

The natural history of the severe form of Hunter's syndrome: a study based on 52 cases.

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years.

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

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

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Enzyme replacement therapy in orphan and ultra-orphan diseases: the limitations of standard economic metrics as exemplified by Fabry-Anderson disease.

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Molecular basis of mucopolysaccharidosis type II in Portugal: identification of four novel mutations.

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Long-term follow-up following bone marrow transplantation for Hunter disease.

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3249184

Enzyme replacement therapy for mucopolysaccharidoses: opinions of patients and families

1 September 2018

1 reference

12 December 2020

Home treatment with Elaprase and Naglazyme is safe in patients with mucopolysaccharidoses types II and VI, respectively

1 reference

12 December 2020

Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI

1 reference

12 December 2020

Home infusion therapy is safe and enhances compliance in patients with mucopolysaccharidoses

1 reference

12 December 2020

Treatment of mucopolysaccharidosis type II (Hunter syndrome) with idursulfase: the relevance of clinical trial end points

1 reference

12 December 2020

Psychosocial problems in Hunter's syndrome

1 reference

12 December 2020

Hydrocephalus in Hunter Syndrome

1 reference

12 December 2020

Mutation analysis in 20 patients with Hunter disease

1 reference

12 December 2020

Intravenous enzyme replacement therapy: better in home or hospital?

1 reference

12 December 2020

Early response to idursulfase treatment in a 3 year-old boy affected of Hunter syndrome

1 reference

12 December 2020

10.1007/S00431-011-1606-3

Identifiers

DOI

1 reference

25 August 2017

1 reference

25 August 2017

PubMed publication ID

22037758

1 reference