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Comment
. 2025 Jul;45(7):435-438.
doi: 10.1007/s40261-025-01443-w. Epub 2025 May 9.

Author's Reply to Perera et al.: A Commentary on "An Early Cost-Utility Model of mRNA-Based Therapies for the Treatment of Methylmalonic and Propionic Acidemia in the United Kingdom"

Pablo E Bretos-Azcona  1 , Matthew Wallace  1 , Murvin Jootun  1 , E Veljanoska  1 , Ion Agirrezabal  1 , Agota Szende  2
Affiliations
Comment

Author's Reply to Perera et al.: A Commentary on "An Early Cost-Utility Model of mRNA-Based Therapies for the Treatment of Methylmalonic and Propionic Acidemia in the United Kingdom"

Pablo E Bretos-Azcona et al. Clin Drug Investig. 2025 Jul.
No abstract available

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Conflict of interest statement

Declarations. Funding: No funding was received for the preparation or publication of this letter. Conflicts of interest: A.S., M.W. and E.V. are employees of Fortrea Inc. P.E.B., M.J. and I.A. were employees of Fortrea Inc. during the conduct of the original study. Availability of data and materials: Not applicable. Ethics approval: Not applicable. Consent to participate: Note applicable. Consent for publication: Not applicable. Code availability: Not applicable. Author contributions: All authors contributed substantially to the preparation of this letter. A.S., M.W. and E.V. were responsible for changes to the existing model and led the interpretation of the results. Each author participated in drafting the letter and performed a critical review, making any required revisions for veracity and intellectual content. All authors have given their final approval to publish this version and accept accountability for its components.

Comment on

References

    1. Perera S, Banerjee G, Sikirica V, et al. Comment on: “An early cost–utility model of mRNA-based therapies for the treatment of methylmalonic and propionic acidemia in the United Kingdom”. Clin Drug Investig. 2025. https://doi.org/10.1007/s40261-025-01442-x .
    1. Bretos-Azcona PE, Wallace M, Jootun M, et al. An early cost–utility model of mRNA-based therapies for the treatment of methylmalonic and propionic acidemia in the United Kingdom. Clin Drug Investig. 2024;44(6):399–412. - PubMed
    1. Li M, Dick A, Montenovo M, et al. Cost-effectiveness of liver transplantation in methylmalonic and propionic acidemias. Liver Transpl. 2015;21(9):1208–18. - PubMed
    1. Quintero J, Molera C, Juamperez J, et al. The role of liver transplantation in propionic acidemia. Liver Transpl. 2018;24(12):1736–45. - PubMed
    1. Chu TH, Chien YH, Lin HY, et al. Methylmalonic acidemia/propionic academia—the biochemical presentation and comparing the outcome between liver transplantation versus non-liver transplantation groups. Orphanet J Rare Dis. 2019;14(1):73. - PubMed - PMC

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