RAS/MAPK pathway modulation with simvastatin in children with Noonan syndrome: a multicentre, randomised, double-blind, placebo-controlled phase 3 trial.

Edouard T, Capri Y, Verloes A, Amouroux C, Bournez M, Bretones P, Coutant R, Houang M, Amsellem Jager J, de Kerdanet M, Lacombe D, Lefevre C, Netchine I, Nicolino M, Pigeon Kherchiche P, Reynaud R, Rossi M, Berot A, Van Gils J, Willems M, Kieffer I, Dulac Y, Prickett T, Espiner E, Gennero I, Auriol F, Lepage B, Yart A

Open source

DOI: 10.1016/j.eclinm.2026.104114
Published: 2026 Sep
Container: EClinicalMedicine
Publisher: Not recorded
Open access: yes

Credibility signals

limited evidence Score 45/100 under policy 1.0.0. This is a metadata assessment, not a judgment of the paper's conclusions.

Show all credibility signals

cautionDOI registered: No matching Crossref record was present in this response. Source: Crossref; license: CC0 metadata
cautionDOI resolves: No matching Crossref record was present in this response. Source: Crossref; license: CC0 metadata
not scoredDirectory of Open Access Journals: No matching DOAJ record was present in this response. No allow-list match; this is not evidence of low credibility. Source: DOAJ; license: CC0
not scoredMEDLINE indexed: Not checked or no result supplied; no credibility inference made. Source: No authority result supplied; license: Unknown
not scoredOpenAlex core source: Not checked or no result supplied; no credibility inference made. Source: No authority result supplied; license: Unknown
not scoredKnown publisher allow-list: Not checked or no result supplied; no credibility inference made. Source: No authority result supplied; license: Unknown
not scoredROR affiliation: Not checked or no result supplied; no credibility inference made. Source: No authority result supplied; license: Unknown
not scoredRetraction Watch retraction: No retraction notice matched this DOI in the deployed snapshot. No matching event found; coverage may be incomplete. Source: Retraction Watch; license: CC BY 4.0
not scoredRetraction Watch expression of concern: No expression of concern notice matched this DOI in the deployed snapshot. No matching event found; coverage may be incomplete. Source: Retraction Watch; license: CC BY 4.0
not scoredRetraction Watch correction: No correction notice matched this DOI in the deployed snapshot. No matching event found; coverage may be incomplete. Source: Retraction Watch; license: CC BY 4.0
not scoredRetraction Watch reinstatement: No reinstatement notice matched this DOI in the deployed snapshot. No matching event found; coverage may be incomplete. Source: Retraction Watch; license: CC BY 4.0
supportingOpen access status: Normalized open-access status: open. Source: Normalized work metadata; license: Caller-provided; provenance license not supplied to scorer
not scoredPublication license: Not checked or no result supplied; no credibility inference made. Source: No authority result supplied; license: Unknown
not scoredPublication version: A publication version was supplied but is not scored. Source: Normalized work metadata; license: Caller-provided; provenance license not supplied to scorer
cautionMetadata completeness: 5 of 6 scored descriptive metadata groups are present; missing fields increase uncertainty. Source: Normalized work metadata; license: Caller-provided; provenance license not supplied to scorer

Cite this work

BibTeX

@article{allodium:10.1016/j.eclinm.2026.104114,
  title = {RAS/MAPK pathway modulation with simvastatin in children with Noonan syndrome: a multicentre, randomised, double-blind, placebo-controlled phase 3 trial.},
  author = {Edouard T and Capri Y and Verloes A and Amouroux C and Bournez M and Bretones P and Coutant R and Houang M and Amsellem Jager J and de Kerdanet M and Lacombe D and Lefevre C and Netchine I and Nicolino M and Pigeon Kherchiche P and Reynaud R and Rossi M and Berot A and Van Gils J and Willems M and Kieffer I and Dulac Y and Prickett T and Espiner E and Gennero I and Auriol F and Lepage B and Yart A},
  year = {2026},
  journal = {EClinicalMedicine},
  doi = {10.1016/j.eclinm.2026.104114},
  url = {https://doi.org/10.1016/j.eclinm.2026.104114}
}

RIS

TY  - JOUR
TI  - RAS/MAPK pathway modulation with simvastatin in children with Noonan syndrome: a multicentre, randomised, double-blind, placebo-controlled phase 3 trial.
AU  - Edouard T
AU  - Capri Y
AU  - Verloes A
AU  - Amouroux C
AU  - Bournez M
AU  - Bretones P
AU  - Coutant R
AU  - Houang M
AU  - Amsellem Jager J
AU  - de Kerdanet M
AU  - Lacombe D
AU  - Lefevre C
AU  - Netchine I
AU  - Nicolino M
AU  - Pigeon Kherchiche P
AU  - Reynaud R
AU  - Rossi M
AU  - Berot A
AU  - Van Gils J
AU  - Willems M
AU  - Kieffer I
AU  - Dulac Y
AU  - Prickett T
AU  - Espiner E
AU  - Gennero I
AU  - Auriol F
AU  - Lepage B
AU  - Yart A
PY  - 2026
JO  - EClinicalMedicine
DO  - 10.1016/j.eclinm.2026.104114
UR  - https://doi.org/10.1016/j.eclinm.2026.104114
ER  -

APA

T, E., Y, C., A, V., C, A., M, B., P, B., R, C., M, H., J, A. J., M, D. K., D, L., C, L., I, N., M, N., P, P. K., R, R., M, R., A, B., J, V. G., M, W., I, K., Y, D., T, P., E, E., I, G., F, A., B, L., & A, Y. (2026). RAS/MAPK pathway modulation with simvastatin in children with Noonan syndrome: a multicentre, randomised, double-blind, placebo-controlled phase 3 trial.. EClinicalMedicine. https://doi.org/10.1016/j.eclinm.2026.104114

Source records

pubmed · retrieved 2026-09-26T11:15:14.011Z