Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study

@article{Anderson2014EffectivenessOE,
  title={Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study},
  author={Larry J. Anderson and William Edward Henley and Kenneth M Wyatt and Vassilki Nikolaou and Stephen Waldek and Derralynn Hughes and Robin H L Lachmann and Sarah B Logan},
  journal={Journal of Inherited Metabolic Disease},
  year={2014},
  volume={37},
  pages={945-952}
}
To determine the effectiveness of enzyme replacement therapy (ERT) for adults with late-onset Pompe disease. A longitudinal cohort study including prospective and retrospective clinical outcome data. Age- and gender-adjusted treatment effects were estimated using generalised linear mixed models. Treated patients contributed data before and during treatment. Untreated patients contributed natural history data. Consenting adults (N = 62) with a diagnosis of late-onset Pompe disease who attended a… CONTINUE READING
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Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study

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  • Orphanet J Rare Dis
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