Evidence map›Paper›PMID 40843507›Full record

Trial reportJournal of comparative effectiveness research2025

Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial.

Dmitry Vlodavets, Shiwen Wu, Anna Kostera-Pruszczyk, Jong-Hee Chae, Sheffali Gulati, Jin-Hong Shin, Michelle Lorentzos, Anita Cairns, Yuh-Jyh Jong, Hirofumi Komaki and 12 more

Abstract readClinical Trial, Phase IIIRandomized Controlled TrialMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of comparative effectiveness research, 2025. 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
–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

1 citing paper in PubMed.

  1. RiboScreenBiomedicines · 2026
    Review
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

22 authors.

Dmitry VlodavetsRussian Children Neuromuscular Center, Veltischev Clinical Pediatric & Pediatric Surgery Research Institute of Pirogov Russian National Research Medical University, Moscow, Russia.ORCID 0000-0003-2635-2752
Shiwen WuThe Third Medical Center of PLA General Hospital, Beijing, China.
Anna Kostera-PruszczykDepartment of Neurology, Medical University of Warsaw, Poland.
Jong-Hee ChaeSeoul National University Hospital, Seoul, South Korea.
Sheffali GulatiAll India Institute of Medical Sciences, New Delhi, India.
Jin-Hong ShinDepartment of Neurology, Pusan National University School of Medicine, Yangsan, South Korea.
Michelle LorentzosThe Children's Hospital at Westmead, Westmead, New South Wales, Australia.
Anita CairnsDepartment of Neurosciences, Queensland Children's Hospital, South Brisbane, Queensland, Australia.
Yuh-Jyh JongGraduate Institute of Clinical Medicine, College of Medicine, Kaohsiung Medical University, & Departments of Pediatrics & Laboratory Medicine, & Translational Research Center of Neuromuscular Diseases, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan.
Hirofumi KomakiTranslational Medical Center, National Center of Neurology & Psychiatry, Tokyo, Japan.
Jeffrey StatlandUniversity of Kansas Medical Center, Kansas City, KS, USA.
Alexandra Prufer de Queiroz Campos AraujoDepartment of Pediatrics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Juliana Gurgel-GiannettiDepartment of Pediatrics, Faculty of Medicine, Federal University of Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
Yasuhiro TakeshimaDepartment of Pediatrics, Hyogo Medical University, Nishinomiya, Japan.
Rosa E Escobar CedilloInstituto Nacional de Rehabilitacion, Mexico City, Mexico.
Peter KarachunskiUniversity of Minnesota, Minneapolis, MN, USA.
Kazuhiro HaginoyaDepartment of Pediatric Neurology, Miyagi Children's Hospital, Sendai, Japan.
Vinay PenematsaPTC Therapeutics Inc., Warren, NJ, USA.
Connie ChouPTC Therapeutics Inc., Warren, NJ, USA.
Paula WilliamsPTC Therapeutics Inc., Warren, NJ, USA.
Christian WernerPTC Therapeutics Germany GmbH, Frankfurt, Germany.
Craig M McDonaldUniversity of California Davis School of Medicine, Sacramento, CA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

Muscular Dystrophy, DuchenneOxadiazolesAdolescentChildDouble-Blind MethodHumansMaleTreatment OutcomeWalk TestatalurenOxadiazoles6-minute walk distanceatalurenefficacyloss of ambulationnonsense mutation Duchenne muscular dystrophysafetystand from supineStudy 041

Identifiers

PMID40843507
PMCPMC12465948

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.