SIMD 2024

Poster 147: Pegtibatinase, An Investigational Enzyme Replacement Therapy, for the Treatment of Classical Homocystinuria (HCU): Design of the HARMONY Phase 3 Study

Janet Thomas, Tawfeg Ben-Omran, Harvey Levy, Francois Maillot, Sharon McDermott, Sergio Slawka, Can Ficicioglu

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