Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature
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Title
Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature
Authors
Keywords
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Journal
GENETICS IN MEDICINE
Volume -, Issue -, Pages -
Publisher
Springer Science and Business Media LLC
Online
2020-01-06
DOI
10.1038/s41436-019-0738-0
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Related references
Note: Only part of the references are listed.- Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy
- (2018) Rossella Parini et al. Orphanet Journal of Rare Diseases
- Longitudinal follow-up to evaluate speech disorders in early-treated patients with infantile-onset Pompe disease
- (2017) Yin-Ting Zeng et al. EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY
- Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study
- (2016) C. M. van Gelder et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Very Early Treatment for Infantile-Onset Pompe Disease Contributes to Better Outcomes
- (2016) Chia-Feng Yang et al. JOURNAL OF PEDIATRICS
- Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years
- (2016) Karolina M. Stepien et al. MOLECULAR GENETICS AND METABOLISM
- Slow, progressive myopathy in neonatally treated patients with infantile-onset Pompe disease: a muscle magnetic resonance imaging study
- (2016) Steven Shinn-Forng Peng et al. Orphanet Journal of Rare Diseases
- Long-Term Prognosis of Patients with Infantile-Onset Pompe Disease Diagnosed by Newborn Screening and Treated since Birth
- (2015) Yin-Hsiu Chien et al. JOURNAL OF PEDIATRICS
- Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease
- (2015) Laura E. Case et al. NEUROMUSCULAR DISORDERS
- An official European Respiratory Society/American Thoracic Society technical standard: field walking tests in chronic respiratory disease
- (2014) Anne E. Holland et al. EUROPEAN RESPIRATORY JOURNAL
- Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy
- (2013) Sean N Prater et al. Orphanet Journal of Rare Diseases
- Reference values for the 6-minute walk test in healthy children and adolescents in Switzerland
- (2013) Silvia Ulrich et al. BMC Pulmonary Medicine
- The emerging phenotype of long-term survivors with infantile Pompe disease
- (2012) Sean N. Prater et al. GENETICS IN MEDICINE
- A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
- (2010) Ans T. van der Ploeg et al. NEW ENGLAND JOURNAL OF MEDICINE
- Maximal Tongue Strength in Typically Developing Children and Adolescents
- (2009) Nancy L. Potter et al. DYSPHAGIA
- Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease
- (2009) Marc Nicolino et al. GENETICS IN MEDICINE
- Long-term monitoring of patients with infantile-onset Pompe disease on enzyme replacement therapy using a urinary glucose tetrasaccharide biomarker
- (2009) Sarah P Young et al. GENETICS IN MEDICINE
- Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants
- (2009) Priya S. Kishnani et al. MOLECULAR GENETICS AND METABOLISM
- Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease
- (2009) Priya S Kishnani et al. PEDIATRIC RESEARCH
- Pompe Disease in Infants: Improving the Prognosis by Newborn Screening and Early Treatment
- (2009) Y.-H. Chien et al. PEDIATRICS
- The 6-minute walk test: normal values for children of 4-11 years of age
- (2007) A E Lammers et al. ARCHIVES OF DISEASE IN CHILDHOOD
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