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Biotechnology & Biotechnological Equipment Volume 38, 2024 - Issue 1
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Letter to the Editor

Letter to the Editor concerning ‘cost-effectiveness of velmanase alfa vs. bone marrow transplantation or no causal therapy in patients with mild to moderate alpha-mannosidosis’

A. L. Stevensona Health Economics and Outcomes Research, Global Rare Diseases, Chiesi Ltd, Manchester, UKCorrespondence[email protected]
View further author information
,
S. Chang-Douglassb Modeling & Simulation, Evidence Synthesis, Modeling and Communication, Evidera Ltd, London, UKView further author information
,
P. Kazmierskab Modeling & Simulation, Evidence Synthesis, Modeling and Communication, Evidera Ltd, London, UKView further author information
,
A. Benedictc Scientific Affairs, Modeling & Simulation, Evidence Synthesis, Modeling and Communication, Evidera Ltd, Budapest, HungaryView further author information
,
K. Azimpourd Health Economics and Outcome Research, Chiesi Canada Corp, Montreal, CanadaView further author information
&
P. Dorlinge Head of Health Economics Outcome Research & Real World Evidence, Rare Diseases, Chiesi USA, Inc, Boston, MA, USAView further author information
Article: 2386487 | Published online: 01 Aug 2024

References

  • Antanasković A, Stević I, Gojak R, et al. Cost-effectiveness of velmanase alfa vs. bone marrow transplantation or no causal therapy in patients with mild to moderate alpha-mannosidosis. Biotechnol Biotechnol Equip. 2023;37(1):2271574. doi:10.1080/13102818.2023.2271574.
  • Borgwardt L, Guffon N, Amraoui Y, et al. Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial. J Inherit Metab Dis. 2018;41(6):1215–1223. doi:10.1007/s10545-018-0185-0.
  • Lund AM, Borgwardt L, Cattaneo F, et al. Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (Velmanase alfa) treatment in patients with alpha-mannosidosis. J Inherit Metab Dis. 2018;41(6):1225–1233. doi:10.1007/s10545-018-0175-2.
  • Guffon N, editor Longterm efficacy of velmanase alfa treatment in patients with alphamannosidosis: pooled data from two extension studies (up to 12 years of therapy). 20th Annual WORLD Symposium; 2024 Feb 4–9; San Diego, CA.
  • Lipiński P, Różdżyńska-Świątkowska A, Iwanicka-Pronicka K, et al. Long-term outcome of patients with alpha-mannosidosis–a single center study. Mol Genet Metab Rep. 2022;30:100826. doi:10.1016/j.ymgmr.2021.100826.
  • Hennermann JB, Raebel EM, Donà F, et al. Mortality in patients with alpha-mannosidosis: a review of patients’ data and the literature. Orphanet J Rare Dis. 2022;17(1):287. doi:10.1186/s13023-022-02422-6.
  • National Institute for Health and Care Excellence (NICE). Velmanase alfa for treating alpha-mannosidosis: NICE technology appraisal guidance HST29 2023; [cited 2024 Feb 28]. Available from: https://www.nice.org.uk/guidance/hst29.
  • Guffon N, Gaches F, Daridon C, Heron-Longe B, editors. Long-term efficacy of velmanase alfa in patients with alpha-mannosidosis: retrospective analysis of a Frenchregistry for up to 9.5 years. SSIEM Annual Symposium. Frieburg (Germany): Journal of Inherited Metabolic Disease; 2022.

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