Evidence map›Paper›PMID 42250073›Full record

ArticleAdvances in therapy2026

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

Melissa Lah, Jessica Kopesky, Erika Vucko

Erratum issuedAbstract readEditorial
PubMed Publisher
In one paragraph

Article in Advances in therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

3 authors.

Melissa LahDepartment of Medical and Molecular Genetics, Indiana University School of Medicine/IUH, 975 W. Walnut St, IB130, Indianapolis, IN, 46202, USA. mspurr@iu.edu.ORCID http://orcid.org/0000-0002-2406-8961
Jessica KopeskyDepartment of Clinical Nutrition, Children's Wisconsin, Milwaukee, WI, USA.ORCID http://orcid.org/0009-0008-5030-683X
Erika VuckoDivision of Genetics, Genomics, and Metabolism, Ann & Robert Lurie Children's Hospital of Chicago, Chicago, IL, USA.ORCID http://orcid.org/0000-0003-0143-9675

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Phenylketonuria (PKU) is a rare, autosomal recessive inborn error of metabolism caused by genetic variants of the PAH gene, which encodes phenylalanine hydroxylase (PAH). In PKU, the PAH enzyme has reduced affinity for its substrate [phenylalanine (Phe)] or cofactor [tetrahydrobiopterin (BH

Indexed as

Mechanism of actionPegvaliasePhenylalaninePhenylketonuriaSapropterinSepiapterin

Identifiers

PMID42250073

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.