The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus
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Title
The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus
Authors
Keywords
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Journal
EUROPEAN JOURNAL OF PEDIATRICS
Volume 171, Issue 1, Pages 181-188
Publisher
Springer Nature
Online
2011-10-28
DOI
10.1007/s00431-011-1606-3
References
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Related references
Note: Only part of the references are listed.- Treatment of mucopolysaccharidosis type II (Hunter syndrome) with idursulfase: the relevance of clinical trial end points
- (2011) Emma Glamuzina et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Identification of iduronate sulfatase gene alterations in 70 unrelated Hunter patients
- (2010) Roseline Froissart et al. CLINICAL GENETICS
- THE NATURAL HISTORY OF THE SEVERE FORM OF HUNTER'S SYNDROME: A STUDY BASED ON 52 CASES
- (2010) I. D. Young et al. DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
- 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
- Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI
- (2009) M. Schlander et al. CURRENT MEDICAL RESEARCH AND OPINION
- Bone Marrow Transplantation in Children with Hunter Syndrome: Outcome after 7 to 17 Years
- (2009) Nathalie Guffon et al. JOURNAL OF PEDIATRICS
- Home infusion therapy is safe and enhances compliance in patients with mucopolysaccharidoses
- (2009) Barbara K. Burton et al. MOLECULAR GENETICS AND METABOLISM
- Multidisciplinary Management of Hunter Syndrome
- (2009) J. Muenzer et al. PEDIATRICS
- Transplant Outcomes in Mucopolysaccharidoses
- (2009) Vinod K. Prasad et al. SEMINARS IN HEMATOLOGY
- Early response to idursulfase treatment in a 3 year-old boy affected of Hunter syndrome
- (2008) Enrique Galán-Gómez et al. European Journal of Medical Genetics
- Home treatment with Elaprase® and Naglazyme® is safe in patients with mucopolysaccharidoses types II and VI, respectively
- (2008) S. Bagewadi et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Enzyme Replacement Therapy for Mucopolysaccharidoses: Opinions of Patients and Families
- (2008) David J. Coman et al. JOURNAL OF PEDIATRICS
- 47. A home infusion protocol for MPS II patients on enzyme replacement therapy (ERT)
- (2008) Rachel Katz et al. MOLECULAR GENETICS AND METABOLISM
- 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
- (2008) Paul Harmatz et al. MOLECULAR GENETICS AND METABOLISM
- Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome)
- (2008) R. Martin et al. PEDIATRICS
- Mucopolysaccharidosis I: Management and Treatment Guidelines
- (2008) J. Muenzer et al. PEDIATRICS
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