Shortcast: Screening data for 19 patients with late-onset Pompe disease for a phase I clinical trial

JIMD Podcasts

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