SSIEM 2024

Poster: Pegtibatinase, an Investigational Enzyme Replacement Therapy for Classical Homocystinuria (HCU): Design of the HARMONY and ENSEMBLE Phase 3 Studies

François Maillot; Janet Thomas; Tawfeg Ben-Omran; Harvey Levy; Stephan vom Dahl; Clint Lovell; Sagar A. Vaidya; Can Ficicioglu

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