Evidence map›Paper›PMID 38406379›Full record

ArticleActa myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology2023

Continuitiy of care with ataluren in Duchenne Muscular Dystrophy patients with nonsense mutations after loss of ambulation. Personal experience.

Carlotta Spagnoli, Rachele Adorisio, Luca Bello, Adele D'Amico, Maria Grazia D'Angelo, Marika Pane, Martina Penzo, Pietro Riguzzi, Valeria Sansone, Andrea Vianello and 1 more

Open access · greenAbstract read
In one paragraph

Article in Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology, 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
–field-weighted citation impact, top 64% 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, 0 citations in OpenAlex.

  1. Transition and management of patients with Duchenne Muscular Dystrophy: a narrative review based on Italian experts' opinion and real-world experience.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2024
    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

11 authors at 5 institutions in 1 country.

Carlotta SpagnoliChild Neurology Unit, Presidio Ospedaliero Provinciale Santa Maria Nuova, AUSL-IRCCS di Reggio Emilia, Reggio Emilia, Italy.
Rachele AdorisioHeart Failure, Transplant and Mechanical Cardiocirculatory Support Unit, Department of Pediatric Cardiology and Cardiac Surgery, Heart Lung Transplantation, ERN GUARD HEART: Bambino Gesù Hospital and Research Institute, Rome, Italy.
Luca BelloDepartment of Neurosciences, University of Padua, Padova, Italy.
Adele D'AmicoDepartment of Neuroscience, Catholic University, Rome, Italy.
Maria Grazia D'AngeloIstituto di Ricerca e Cura a Carattere Scientifico E. Medea, La Nostra Famiglia, Bosisio Parini, Italy.
Marika PanePediatric Neurology and Nemo Clinical Centre, Fondazione Policlinico Universitario A. Gemelli IRCSS, Università Cattolica del Sacro Cuore, Roma, Italy.
Martina PenzoDepartment of Neurosciences, University of Padua, Padova, Italy.
Pietro RiguzziDepartment of Neurosciences, University of Padua, Padova, Italy.
Valeria SansoneCentro Clinico Nemo Milan, Neurorehabilitation Unit, Dipartimento di Scienze Biomediche della Salute, University of Milan, Milan, Italy.
Andrea VianelloRespiratory Pathophysiology Division, University of Padua, Padua, Italy.
Carlo FuscoChild Neurology Unit, Presidio Ospedaliero Provinciale Santa Maria Nuova, AUSL-IRCCS di Reggio Emilia, Reggio Emilia, Italy.
University of Padua · ITPresidio Ospedaliero · ITBambino Gesù Children's Hospital · ITUniversità Cattolica del Sacro Cuore · ITUniversity of Milan · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Duchenne Muscular Dystrophy (DMD) includes predictable phases requiring dedicated standard treatments. Therapeutic strategies feature corticosteroids or the more recent gene therapy/stop codon read-through. Ataluren (Translarna

Indexed as

Muscular Dystrophy, DuchenneAdolescentAdultCodon, NonsenseDystrophinHumansOxadiazolesWalkingYoung AdultatalurenCodon, NonsenseDystrophinOxadiazolesatalurenDuchenne Muscular Dystrophyloss of ambulationnonsense mutations

Identifiers

PMID38406379
PMCPMC10883323
OpenAlexW4394768636

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.