Evidence map›Paper›PMID 42306244›Full record

ArticleJIMD reports2026

Long-Term Efficacy and Safety of Glycerol Phenylbutyrate in Japanese Patients With Urea Cycle Disorders: Results From a Phase 3 Switch-Over and 12-Month Extension Study.

Yoichi Wada, Mahoko Furujo, Kenichi Kashimada, Takashi Hamazaki, Hiromi Nyuzuki, Keiko Ichimoto, Toshihiko Kakiuchi, Shirou Matsumoto, Yoriko Watanabe, Chiho Ono and 3 more

Abstract read
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In one paragraph

Article in JIMD reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. 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

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

13 authors.

Yoichi WadaDepartment of Medical Science and Innovation SiRIUS Institute of Medical Research Sendai Japan.ORCID https://orcid.org/0000-0002-2188-5608
Mahoko FurujoDepartment of Pediatrics Okayama Medical Center Okayama Japan.
Kenichi KashimadaDivision of Endocrinology and Metabolism National Center for Child Health and Development Tokyo Japan.ORCID https://orcid.org/0000-0003-2505-5932
Takashi HamazakiDepartment of Pediatrics Osaka Metropolitan University Osaka Japan.
Hiromi NyuzukiDepartment of Pediatrics Niigata University Medical and Dental Hospital Niigata Japan.ORCID https://orcid.org/0000-0003-1502-0875
Keiko IchimotoDepartment of Metabolism Chiba Children's Hospital Chiba Japan.ORCID https://orcid.org/0000-0002-7152-6240
Toshihiko KakiuchiDepartment of Pediatrics Saga University Saga Japan.ORCID https://orcid.org/0000-0002-9995-5522
Shirou MatsumotoDepartment of Pediatrics Kumamoto University Kumamoto Japan.
Yoriko WatanabeDepartment of Pediatrics and Child Health Kurume University Kurume Japan.
Chiho OnoOrphanPacific Inc. Tokyo Japan.
Takako ShimizuOrphanPacific Inc. Tokyo Japan.
Hiroiku FurukawaOrphanPacific Inc. Tokyo Japan.
Kimitoshi NakamuraDepartment of Pediatrics Kumamoto University Kumamoto Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Sodium phenylbutyrate (NaPBA) is used for nitrogen scavenging in urea cycle disorders (UCDs), but its volume, palatability, and sodium load affect adherence and ammonia control. Glycerol phenylbutyrate (GPB) offers an alternative option with demonstrated improvements in metabolic control and palatability. For a Japanese perspective, we undertook an open-label, multicentre, prospective Phase 3 study of efficacy, pharmacokinetics (PK), and safety. In a switch-over, 15 patients received NaPBA for 7 days, then GPB for 7 days. Primary endpoint: 24-h blood ammonia AUC (AUC

Indexed as

ammoniaglycerol phenylbutyrateJapannitrogen scavengerphenylbutyraterare diseaseurea cycle disorder

Identifiers

PMID42306244
PMCPMC13265243

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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.