Correction: Sepiapterin: A Distinct, Dual Mechanism of Action that Leads to Potential Treatment Benefits Across the Spectrum of Phenylketonuria Disease Severities

In the published version, under the Distinct dual mechanism of action of sepiapterin section, the text beginning from “These interactions include the formation of a hydrogen bond with the Tyr325 residue at the active site of the enzyme, which plays an important role in the conversion of Phe to tyrosine (PTC Therapeutics. Data on file. 2023: submitted for publication) [29, 33, 35].” corrected to “These interactions include the formation of a hydrogen bond with the Tyr325 residue at the active site of the enzyme, which plays an important role in the conversion of Phe to tyrosine (PTC Therapeutics. Data on file. 2023: submitted for publication) [35,36].” In Table 2, under the heading APHENITY, “Patients with PKU aged ≥ 2 years in part 1 (N = 157) and part 2 (n = 98)” corrected to “Patients with PKU in part 1 (N = 157) and aged ≥ 2 years in part 2 (n = 98 [primary analysis set])”. In the heading Phase 3 data: APHENITY study, the text beginning from “In part 2 (placebo-controlled, randomized assessment in participants aged ≥ 2 years who had responded to sepiapterin treatment with a ≥ 15% reduction in blood Phe in part 1), there was a significant reduction in blood Phe levels from baseline to week 6 in participants receiving sepiapterin (n = 49) versus placebo (n = 49)” corrected to “In part 2 (placebo-controlled, randomized assessment in participants aged ≥ 2 years who had responded to sepiapterin treatment with a ≥ 15% reduction in blood Phe in part 1), there was a significant reduction in blood Phe levels from baseline to week 6 in participants receiving sepiapterin (n = 49) versus placebo (n = 49) in the primary analysis set (participants with a ≥ 30% reduction in blood Phe in part 1)”. In the heading Phase 3 data: AMPLIPHY study, the text beginning from “with a relative 100% greater reduction (PTC Therapeutics. Data on file. 2025)” corrected to “with a relative 79.2% greater reduction (PTC Therapeutics. Data on file. 2025)”. In the heading, Phase 3 data: open-label long-term extension study, the text “..and in those shown to be previously nonresponsive to BH4 (mean increase of 127% [n = 121])” corrected to “…and in those shown to be previously nonresponsive to BH4 (mean increase of 127% [n = 21])”. In the heading, Phase 3 data: open-label long-term extension study, the text “The most common AEs with sepiapterin (≥ 2% and greater than placebo in the phase 3 APHENITY study) were diarrhea, headache, abdominal pain, hyperphenylalaninemia, feces discoloration, and oropharyngeal pain” corrected to “The most common AEs with sepiapterin (≥ 2% and greater than placebo in the phase 3 APHENITY study) were diarrhea, headache, abdominal pain, hypophenylalaninemia, feces discoloration, and oropharyngeal pain”. The original article has been corrected.