First-in-human case report: AAV9-hGAA gene therapy for a patient with infantile-onset Pompe disease. https://www.medrxiv.org/content/10.1101/2022.12.22.22283398
First-in-human case report: AAV9-hGAA gene therapy for a patient with infantile-onset Pompe disease. https://www.medrxiv.org/content/10.1101/2022.12.22.22283398