Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)
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Title
Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)
Authors
Keywords
Hunter syndrome, Lysosomal storage disease, Idursulfase, Efficacy, Disease registry
Journal
Orphanet Journal of Rare Diseases
Volume 12, Issue 1, Pages -
Publisher
Springer Nature
Online
2017-10-04
DOI
10.1186/s13023-017-0712-3
References
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Related references
Note: Only part of the references are listed.- Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS)
- (2017) Barbara K. Burton et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry
- (2017) Joseph Muenzer et al. Orphanet Journal of Rare Diseases
- Clinical response to long term enzyme replacement treatment in children, adolescent and adult patients with Hunter syndrome
- (2015) Jaime Dalmau Serra et al. MEDICINA CLINICA
- Diagnosis, quality of life, and treatment of patients with Hunter syndrome in the French healthcare system: a retrospective observational study
- (2015) Nathalie Guffon et al. Orphanet Journal of Rare Diseases
- Clinical efficacy of Enzyme Replacement Therapy in paediatric Hunter patients, an independent study of 3.5 years
- (2014) Rosella Tomanin et al. Orphanet Journal of Rare Diseases
- A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4–7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy
- (2013) Roberto Giugliani et al. GENETICS IN MEDICINE
- Clinical and Ultrasonographic Measurement of Liver Size in Normal Children
- (2013) Puja Amatya et al. INDIAN JOURNAL OF PEDIATRICS
- The effect of idursulfase on growth in patients with Hunter syndrome: Data from the Hunter Outcome Survey (HOS)
- (2013) Simon A. Jones et al. MOLECULAR GENETICS AND METABOLISM
- The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5years and older treated with intravenous idursulfase
- (2013) A.J. Barbier et al. MOLECULAR GENETICS AND METABOLISM
- Early initiation of enzyme replacement therapy for the mucopolysaccharidoses
- (2013) Joseph Muenzer MOLECULAR GENETICS AND METABOLISM
- Enzyme replacement therapy for mucopolysaccharidosis II from 3 months of age: a 3-year follow-up
- (2011) A Tylki- Szymanska et al. ACTA PAEDIATRICA
- Idursulfase treatment of Hunter syndrome in children younger than 6 years: Results from the Hunter Outcome Survey
- (2011) Joseph Muenzer et al. GENETICS IN MEDICINE
- Hearing loss in patients with mucopolysaccharidosis II: Data from HOS – the Hunter Outcome Survey
- (2011) Annerose Keilmann et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management
- (2011) Elizabeth A. Braunlin et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Prevalence and Characterization of Cardiac Involvement in Hunter Syndrome
- (2011) Christoph Kampmann et al. JOURNAL OF PEDIATRICS
- Incidence and timing of infusion-related reactions in patients with mucopolysaccharidosis type II (Hunter syndrome) on idursulfase therapy in the real-world setting: A perspective from the Hunter Outcome Survey (HOS)
- (2011) Barbara K. Burton et al. MOLECULAR GENETICS AND METABOLISM
- Therapy for the mucopolysaccharidoses
- (2011) V. Valayannopoulos et al. RHEUMATOLOGY
- Importance of surgical history in diagnosing mucopolysaccharidosis type II (Hunter syndrome): Data from the Hunter Outcome Survey
- (2010) Nancy J. Mendelsohn et al. GENETICS IN MEDICINE
- Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome
- (2010) Joseph Muenzer et al. GENETICS IN MEDICINE
- Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II — data from the Hunter Outcome Survey
- (2010) Barbara K. Burton et al. MOLECULAR GENETICS AND METABOLISM
- Initial report from the Hunter Outcome Survey
- (2009) J Edmond Wraith et al. GENETICS IN MEDICINE
- Mortality and cause of death in mucopolysaccharidosis type II—a historical review based on data from the Hunter Outcome Survey (HOS)
- (2009) S. A. Jones et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Japan Elaprase® Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)
- (2009) Torayuki Okuyama et al. MOLECULAR GENETICS AND METABOLISM
- Multidisciplinary Management of Hunter Syndrome
- (2009) J. Muenzer et al. PEDIATRICS
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