Shortcast: Screening data for 19 patients with late-onset Pompe disease for a phase I clinical trial
Nov 17, 2023•4 min
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Episode description
Dr Will Heath describes insights form screening data derived from 19 patients enrolled in a phase 1 study in late-onset Pompe disease.
Screening data from 19 patients with late-onset Pompe disease for a phase I clinical trial of AAV8 vector-mediated gene therapy
William B. Hannah, et al
https://doi.org/10.1002/jmd2.12391
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