Treatment of mucopolysaccharidosis type II (Hunter syndrome): a Delphi derived practice resource of the American College of Medical Genetics and Genomics (ACMG)
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Title
Treatment of mucopolysaccharidosis type II (Hunter syndrome): a Delphi derived practice resource of the American College of Medical Genetics and Genomics (ACMG)
Authors
Keywords
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Journal
GENETICS IN MEDICINE
Volume -, Issue -, Pages -
Publisher
Springer Science and Business Media LLC
Online
2020-08-03
DOI
10.1038/s41436-020-0909-z
References
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Related references
Note: Only part of the references are listed.- Diagnosis and follow-up of patients with Hunter syndrome in Spain
- (2018) Luis González-Gutiérrez-Solana et al. MEDICINE
- Treatment of mucopolysaccharidosis type II (Hunter syndrome): results from a systematic evidence review
- (2017) Linda A Bradley et al. GENETICS IN MEDICINE
- 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
- Is There a Consensus on Consensus Methodology? Descriptions and Recommendations for Future Consensus Research
- (2016) Jane Waggoner et al. ACADEMIC MEDICINE
- Targeted approaches to induce immune tolerance for Pompe disease therapy
- (2016) Phillip A Doerfler et al. Molecular Therapy-Methods & Clinical Development
- Structural Basis of Mucopolysaccharidosis Type II and Construction of a Database of Mutant Iduronate 2-Sulfatases
- (2016) Seiji Saito et al. PLoS One
- A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II
- (2015) Joseph Muenzer et al. GENETICS IN MEDICINE
- Guidelines for diagnosis and treatment of Hunter Syndrome for clinicians in Latin America
- (2014) Roberto Giugliani et al. GENETICS AND MOLECULAR BIOLOGY
- Defining consensus: A systematic review recommends methodologic criteria for reporting of Delphi studies
- (2014) Ivan R. Diamond et al. JOURNAL OF CLINICAL EPIDEMIOLOGY
- Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series
- (2014) Christina Lampe et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT
- (2013) Suhrad G. Banugaria et al. PLoS One
- The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus
- (2011) Joseph Muenzer et al. EUROPEAN JOURNAL OF PEDIATRICS
- Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease
- (2011) Maurizio Scarpa et al. Orphanet Journal of Rare Diseases
- Initial report from the Hunter Outcome Survey
- (2009) J Edmond Wraith et al. GENETICS IN MEDICINE
- Multidisciplinary Management of Hunter Syndrome
- (2009) J. Muenzer et al. PEDIATRICS
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