Evidence map›Paper›PMID 40434600›Full record

ReviewDrugs in R&D2025

Opinion of the Italian Association of Myology on Ataluren for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy.

Luca Bello, Pietro Riguzzi, Emilio Albamonte, Guja Astrea, Roberta Battini, Andrea Barp, Angela L Berardinelli, Enrico S Bertini, Noemi Brolatti, Claudio Bruno and 31 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Drugs in R&D, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Nonsense-mediated mRNA decay: friend or foe in cancer biology?Cell communication and signaling : CCS · 2026
    Review
  2. Article
  3. Article
  4. 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

41 authors.

Luca BelloDepartment of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, via Giustiniani, 5, 35121, Padua, Italy. luca.bello@unipd.it.ORCID http://orcid.org/0000-0002-3075-6525
Pietro RiguzziDepartment of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, via Giustiniani, 5, 35121, Padua, Italy.
Emilio AlbamonteThe NeMO Center in Milan, Neurorehabilitation Unit, ASST Niguarda Hospital, University of Milan, Milan, Italy.
Guja AstreaDepartment of Neuroscience, IRCCS Stella Maris Foundation, Pisa, Italy.
Roberta BattiniDepartment of Neuroscience, IRCCS Stella Maris Foundation, Pisa, Italy.
Andrea BarpAzienda Provinciale Servizi Sanitari (APSS) Trento, Trento, Italy.
Angela L BerardinelliC. Mondino Foundation, Pavia, Italy.
Enrico S BertiniDepartment of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Noemi BrolattiIRCCS Istituto Giannina Gaslini, Genoa, Italy.
Claudio BrunoIRCCS Istituto Giannina Gaslini, Genoa, Italy.
Stefania CortiFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Adele D'AmicoDepartment of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy.
Maria Grazia D'AngeloScientific Institute IRCCS Eugenio Medea, Bosisio Parini, Italy.
Gianfranco DallavalleAzienda Sanitaria Locale 4 Chiavarese, Chiavari, Italy.
Rocco LiguoriIRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy.
Lorenzo MaggiFondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Francesca MagriFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Michelangelo MancusoCentro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Riccardo MassonFondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Eugenio MercuriCentro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Carlo MinettiIRCCS Istituto Giannina Gaslini, Genoa, Italy.
Sonia MessinaDepartment of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Tiziana MonginiNeuromuscular Unit, Department of Neurosciences RLM, University of Turin, Turin, Italy.
Olimpia MusumeciDepartment of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
Vincenzo NigroMedical Genetics and Cardiomyology, University of Campania "Luigi Vanvitelli", Naples, Italy.
Marika PaneCentro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Chiara PanicucciIRCCS Istituto Giannina Gaslini, Genoa, Italy.
Marina PedemonteIRCCS Istituto Giannina Gaslini, Genoa, Italy.
Antonella PiniIRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy.
Luisa PolitanoMedical Genetics and Cardiomyology, University of Campania "Luigi Vanvitelli", Naples, Italy.
Stefano PrevitaliNeuromuscular Repair Unit, Institute of Experimental Neurology (InSpe), Division of Neuroscience, IRCCS Ospedale San Raffaele and Vita-Salute San Raffaele University, Milan, Italy.
Federica RicciNeuromuscular Unit, Department of Neurosciences RLM, University of Turin, Turin, Italy.
Giulia RicciDepartment of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.
Lucia RuggieroDepartment of Neurosciences, Reproductive and Odontostomatological Sciences, University of Naples "Federico II", Naples, Italy.
Valeria SansoneThe NeMO Center in Milan, Neurorehabilitation Unit, ASST Niguarda Hospital, University of Milan, Milan, Italy.
Gabriele SicilianoDepartment of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.
Antonio TrabaccaScientific Institute IRCCS Eugenio Medea, Bosisio Parini, Italy.
Federica TruccoDepartment of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy.
Daniele VelardoFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Elena PegoraroDepartment of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, via Giustiniani, 5, 35121, Padua, Italy.
Giacomo P ComiFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The Italian Duchenne muscular dystrophy expert clinicians, gathered in the Italian Association of Myology (AIM), intend to express a position against the suspension of the Marketing Authorization of ataluren (Translarna

Indexed as

Codon, NonsenseMuscular Dystrophy, DuchenneOxadiazolesHumansItalyRandomized Controlled Trials as TopicatalurenCodon, NonsenseOxadiazoles

Identifiers

PMID40434600
PMCPMC12185855

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.