Preface
Editorial for focused issue “Pompe disease: from basics to current and emerging therapies”
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2019;
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Original Article
Variable clinical features and genotype-phenotype correlations in 18 patients with late-onset Pompe disease
Annals of Translational Medicine
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Assessing metabolic profiles in human myoblasts from patients with late-onset Pompe disease
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Review Article
Molecular genetics of Pompe disease: a comprehensive overview
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Pros and cons of different ways to address dysfunctional autophagy in Pompe disease
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Restoring the regenerative balance in neuromuscular disorders: satellite cell activation as therapeutic target in Pompe disease
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Newborn screening: Taiwanese experience
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Nutrition and exercise in Pompe disease
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Long-term outcome and unmet needs in infantile-onset Pompe disease
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Multisystem late onset Pompe disease (LOPD): an update on clinical aspects
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Immunological challenges and approaches to immunomodulation in Pompe disease: a literature review
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Diagnostic tools in late onset Pompe disease (LOPD)
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Progress and challenges of gene therapy for Pompe disease
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Liver depot gene therapy for Pompe disease
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An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond
Annals of Translational Medicine
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Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy
Annals of Translational Medicine
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Challenges in treating Pompe disease: an industry perspective
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Pompe disease: what are we missing?
Annals of Translational Medicine
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The role of patient advocacy organizations in shaping medical research: the Pompe model
Annals of Translational Medicine
2019;
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(July 2019)
Disclosure:
The focused issue “Pompe Disease” was commissioned by the editorial office, Annals of Translational Medicine without any sponsorship or funding. Rosa Puertollano and Nina Raben served as the unpaid Guest Editors for the focused issue.
