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Pompe Disease Registry
The objectives of the Registry are: To enhance the understanding of the variability, progression, and natural history of the key manifestations of Pompe disease; To assist the Pompe medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; To characterize …
deficiency
gaa gene
glycogen storage disease type ii
myozyme
replacement therapy
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- 19 Feb, 2024
- 169 locations