Clinical trial design for HMI-203 investigational gene therapy for mucopolysaccharidosis type II (MPS II) informed by cross-correction potential and KOL input

Jacinthe Gingras, Jeff Haroldson, Laura J. Smith,Kruti Patel, Jen Salstrom, Julie Jordan, Gabriel M. Cohn,Omar Francone,Albert Seymour

Molecular Genetics and Metabolism(2022)

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