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A phase I/II open-label gene replacement clinical study for late onset Pompe disease
A phase I/II open-label gene replacement clinical study for late onset Pompe disease
2021
John W. Day
Tahseen Mozaffar
Cheryl Wong Po Foo
Nathan Bachtell
Edward Conner
Keywords:
late onset
Gene replacement
Gastroenterology
open label
Medicine
Disease
Internal medicine
phase i ii
clinical study
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