Comment on: "An Early Cost-Utility Model of mRNA-Based Therapies for the Treatment of Methylmalonic and Propionic Acidemia in the United Kingdom"
- PMID: 40343677
- PMCID: PMC12255541
- DOI: 10.1007/s40261-025-01442-x
Comment on: "An Early Cost-Utility Model of mRNA-Based Therapies for the Treatment of Methylmalonic and Propionic Acidemia in the United Kingdom"
Conflict of interest statement
Declarations. Funding: This work was funded by Moderna, Inc. Conflicts of Interest: SP, GB, VS and EK are employees of Moderna, Inc. EC, CB, and SCB are employees of FIECON, which is funded by Moderna, Inc. SG has served on an advisory board for Moderna, Inc. SY has received honoraria from Moderna, Inc. Ethics Approval: Not applicable. Consent to Participate: Not applicable. Consent for Publication: Not applicable. Availability of Data and Material: Not applicable. Code Availability: Not applicable. Author Contributions: All authors have contributed equally to the development of this commentary. All authors read and approved the final version of the commentary.
Comment on
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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 Jun;44(6):399-412. doi: 10.1007/s40261-024-01363-1. Epub 2024 May 25. Clin Drug Investig. 2024. PMID: 38796677
References
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- 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;44:399–412. - PubMed
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- Waisbren SE. Review of neuropsychological outcomes in isolated methylmalonic acidemia: recommendations for assessing impact of treatments. Metab Brain Dis. 2022;37:1317–35. - PubMed
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- Fujisawa D, Nakamura K, Mitsubuchi H, et al. Clinical features and management of organic acidemias in Japan. J Hum Genet. 2013;58:769–74. - PubMed
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- Hörster F, Baumgartner MR, Viardot C, et al. Long-term outcome in methylmalonic acidurias is influenced by the underlying defect (mut0, mut−, cblA, cblB). Pediatr Res. 2007;62:225–30. - PubMed
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