A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

计划入组 56 人 · 首次发布于 2024 年 3 月 8 日

Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU

计划入组 148 人 · 首次发布于 2026 年 7 月 27 日

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

计划入组 247 人 · 首次发布于 2021 年 12 月 21 日

Sepiapterin(Sephience)

C₉H₁₁N₅O₃

Sepiapterin reductase inhibitors (Immune disorder)

sepiapterin reductase inhibitors (lower back pain)

Sapience Therapeutics, Inc.

United States

PTC Therapeutics' Sephience (sepiapterin) Data Reinforce Broad PKU Benefits, Including Diet Liberalization and Sustained Metabolic Control

PTC Therapeutics' Sephience Receives Positive CHMP Opinion for Phenylketonuria Treatment Across All Age Groups

PTC Therapeutics Secures Global Approvals for Sephience in PKU Treatment Across Canada and Japan