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Jim LaValle, a Texas-based clinical pharmacist and chair of the International Peptide Society, who testified at the hearings, noted that restrictions still exist

Beyond long-standing dietary and vitamin-based management, recent developments include enzyme replacement therapy programs, pharmacological chaperone approaches designed to stabilize the CBS enzyme, and early-stage gene therapy candidates with one prior program (pegtarviliase) discontinued and succeeded by a newer candidate (rebranded SYNT-202) still progressing through development

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However, in the rest of the world, the parenteral route remains the most used