Evidence map›Paper›PMID 38586166›Full record

ArticleActa myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology2024

Treatment with ataluren in four symptomatic Duchenne carriers. A pilot study.

Amir Dori, Marianna Scutifero, Luigia Passamano, Dario Zoppi, Lucia Ruggiero, Antonio Trabacca, Luisa Politano

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. 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

7 authors.

Amir DoriDepartment of Neurology, Chaim Sheba Medical Center, HaShomer, and Joseph Sagol Neuroscience Center, Faculty of Medicine, Aviv University, Aviv, Israel.
Marianna ScutiferoCardiomyology and Medical Genetics, University of Campania Luigi Vanvitelli, Naples, Italy.
Luigia PassamanoCardiomyology and Medical Genetics, University of Campania Luigi Vanvitelli, Naples, Italy.
Dario ZoppiDepartment of Neurosciences, Reproductive and Odontostomatological Sciences, University of Naples "Federico II", Naples, Italy.
Lucia RuggieroDepartment of Neurosciences, Reproductive and Odontostomatological Sciences, University of Naples "Federico II", Naples, Italy.
Antonio TrabaccaScientific Institute IRCCS "E. Medea", Unit for Severe disabilities in developmental age and young adults (Developmental Neurology and Neurorehabilitation), Brindisi, Italy.
Luisa PolitanoCardiomyology and Medical Genetics, University of Campania Luigi Vanvitelli, Naples, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a devastating X-linked neuromuscular disorder caused by dystrophin gene deletions (75%), duplications (15-20%) and point mutations (5-10%), a small portion of which are nonsense mutations. Women carrying dystrophin gene mutations are commonly

Indexed as

CardiomyopathiesMuscular Dystrophy, DuchenneOxadiazolesChild, PreschoolCodon, NonsenseDystrophinFemaleHumansPilot ProjectsatalurenCodon, NonsenseDystrophinOxadiazolesatalurenDMD symptomatic/affected carriersDuchenne muscular dystrophymanifesting carriersnonsense mutations

Identifiers

PMID38586166
PMCPMC10997039

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.