Evidence map›Paper›PMID 40040902›Full record

ArticleERJ open research2025

Efficacy and safety of CFTR modulators in patients with interstitial lung disease caused by ABCA3 transporter deficiency.

Mathilde Le Brun, Nadia Nathan, Camille Louvrier, Marie Legendre, Severine Feuillet, Justine Frija-Masson, Mwetty Onanga, Lise Morer, Marie-Pierre Debray, Isabelle Fajac and 4 more

Abstract read
In one paragraph

Article in ERJ open research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed, 1 pooled it
–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

7 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Old and emerging therapies for childhood interstitial lung disease (chILD): a systematic review.European respiratory review : an official journal of the European Respiratory Society · 2026
    Pooled it
  2. Lentiviral-mediated gene complementation to rescue pathogenic ABCA3 variants.American journal of respiratory cell and molecular biology · 2026
    Article
  3. Article
  4. Lentiviral-mediated gene complementation rescues pathogenicbioRxiv : the preprint server for biology · 2025
    Article
  5. Respirology case reports · 2025
    Article
  6. Review
  7. 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

14 authors.

Mathilde Le BrunService de Pneumologie A, Hôpital Bichat, Assistance Publique-Hôpitaux de Paris (APHP), Paris, France.ORCID https://orcid.org/0009-0007-1906-453X
Nadia NathanSorbonne Université, APHP, Pediatric Pulmonology, Department and Reference Center for Rare Lung Diseases RespiRare, Armand Trousseau Hospital, Paris, France.ORCID https://orcid.org/0000-0001-5149-7975
Camille LouvrierSorbonne Université, APHP, Pediatric Pulmonology, Department and Reference Center for Rare Lung Diseases RespiRare, Armand Trousseau Hospital, Paris, France.ORCID https://orcid.org/0000-0003-2105-1117
Marie LegendreInserm UMR_S933, Laboratory of Childhood Genetic Diseases, Sorbonne Université, Paris, France.ORCID https://orcid.org/0000-0003-2178-0846
Severine FeuilletService de Pneumologie Cancérologie thoracique - hôpital Marie Lannelongue, Le Plessis-Robinson.
Justine Frija-MassonUniversité de Paris, NeuroDiderot, Inserm U1141 APHP, Hôpital Bichat, Explorations Fonctionnelles et Centre du Sommeil - Département de Physiologie Clinique, Paris, France.ORCID https://orcid.org/0000-0001-5575-3913
Mwetty OnangaService de Pneumologie A, Hôpital Bichat, Assistance Publique-Hôpitaux de Paris (APHP), Paris, France.
Lise MorerService de Pneumologie B Hôpital Bichat, APHP, Paris, France.
Marie-Pierre DebrayUniversité Paris Cité, Inserm, Paris, France.ORCID https://orcid.org/0000-0003-2109-5662
Isabelle FajacUniversité Paris Cité, Inserm, Paris, France.
Pierre-Régis BurgelUniversité Paris Cité, Inserm, Paris, France.ORCID https://orcid.org/0000-0003-0903-9828
Sylvain Marchand-AdamService de Pneumologie, Hopital de Tours, Tours, France.ORCID https://orcid.org/0000-0002-9522-3738
Bruno CrestaniService de Pneumologie A, Hôpital Bichat, Assistance Publique-Hôpitaux de Paris (APHP), Paris, France.ORCID https://orcid.org/0000-0002-2961-3455
Raphaël BorieService de Pneumologie A, Hôpital Bichat, Assistance Publique-Hôpitaux de Paris (APHP), Paris, France.ORCID https://orcid.org/0000-0002-9906-0024

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Identifiers

PMID40040902
PMCPMC11874218

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.