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Category: AT845

AT845 Gene Therapy - AAV8 delivery of the human GAA gene to treat Pompe Disease (Image from Audentes Therapeutics)

August 6, 2020

by BJP · Published August 6, 2020 · Last modified March 7, 2021

AT845: Audentes Therapeutics Begins Recruiting Patients for new Phase 1/2 LOPD Gene Therapy Clinical Trial for Pompe Disease (FORTIS)

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AT845: Audentes Therapeutics Begins Recruiting Patients for new Phase 1/2 LOPD Gene Therapy Clinical Trial for Pompe Disease (FORTIS)
Higher dosing of alglucosidase alfa (ERT) improves outcomes in children with Pompe disease: a clinical study and review of the literature
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