Evidence map›Paper›PMID 38053938›Full record

ArticleMolecular genetics and metabolism reports2023

Pharmacodynamics, safety, tolerability and pharmacokinetics of a single oral dose of an engineered phenylalanine ammonia-lyase in patients with phenylketonuria.

Timothy Nicholas Fazio, Louise Healy, Tim Heise, Anita Inwood, Catherine Manolikos, Yusof Rahman, Hans-Juergen Woerle, Christian J Hendriksz

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism reports, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.6field-weighted citation impact, top 33% of its field
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

1 citing paper in PubMed, 3 citations in OpenAlex.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 5 institutions in 3 countries.

Timothy Nicholas FazioRoyal Melbourne Hospital, Melbourne, Victoria, Australia; Melbourne Medical School, University of Melbourne, Parkville, Victoria, Australia.
Louise HealyMetabolic Dietary Disorders Association, PO Box 33, Montrose, Victoria, 3765, Australia.
Tim HeiseProfil, Neuss, Germany.
Anita InwoodQueensland Lifespan Metabolic Medicine Service, Brisbane, Queensland, Australia.
Catherine ManolikosRoyal Perth Hospital, Perth, Australia.
Yusof RahmanDepartment of Genetic Medicine, Westmead Hospital, Sydney, Australia.
Hans-Juergen WoerleNestlé Health Science, Avenue Nestlé 55, 1800, Vevey, Switzerland.
Christian J HendrikszNestlé Health Science, Avenue Nestlé 55, 1800, Vevey, Switzerland.
Nestlé (Switzerland) · CHProfil Institute for Metabolic Research · DERoyal Perth Hospital · AUThe Royal Melbourne Hospital · AUWestmead Hospital · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The cornerstone treatment of hyperphenylalaninemia (HPA) and phenylketonuria (PKU) is a lifelong low-protein diet with phenylalanine (Phe) free L-amino acid supplements. However, the PKU diet has significant shortcomings, and there is a clinically unmet need for new therapeutics to improve patient outcomes. CDX-6114 is a modified phenylalanine ammonia-lyase (PAL) enzyme obtained by a mutation in the

Indexed as

CDX-6114Cinnamic acidEnzymePhenylalanine hydroxylasePhenylketonuria

Identifiers

PMID38053938
PMCPMC10694774
OpenAlexW4387072871

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.