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

Concepedia

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

60

Citations

25

References

2014

Year

Lindsey Anderson, William Henley, Katrina Wyatt, Vasilis Nikolaou, S. Waldek, Derralynn Hughes, Robin Lachmann, Stuart Logan

Journal of Inherited Metabolic Disease

Abstract

The results of this longitudinal cohort study of 62 adults with late-onset Pompe disease, provide further evidence on the effectiveness of ERT in this rare condition.

References

	Year	Citations

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