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. 2025 Jan-Dec;67(1):e70106.
doi: 10.1111/ped.70106.

Potential efficacy of vosoritide for foramen magnum stenosis in a patient with achondroplasia

Mitsuyoshi Suzuki  1 , Koichiro Sakamoto  2 , Yumiko Sakurai  1 , Hiroki Saito  1 , Satoshi Nakano  1 , Naoto Nishizaki  3
Affiliations

Potential efficacy of vosoritide for foramen magnum stenosis in a patient with achondroplasia

Mitsuyoshi Suzuki et al. Pediatr Int. 2025 Jan-Dec.
No abstract available

Keywords: FGFR3 pathway; achondroplasia; foramen magnum stenosis; vosoritide.

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References

REFERENCES

    1. Savarirayan R, Tofts L, Irving M, Wilcox W, Bacino CA, Hoover‐Fong J, et al. Once‐daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double‐blind, phase 3, placebo‐controlled, multicentre trial. Lancet. 2020;396:684–692.
    1. Savarirayan R. Advances in the management of achondroplasia. Nat Rev Endocrinol. 2024;20:443–444.
    1. Cheung MS, Cocca A, Harvey CH, Brett CSS, Offiah AC, Borg S, et al. Natural history of spinal cord compression stage AFMS3 in infants with achondroplasia: retrospective cohort study. Arch Dis Child. 2024;109(12):1025–1028. https://doi.org/10.1136/archdischild‐2024‐327035
    1. Savarirayan R, Wilcox WR, Harmatz P, Phillips J III, Polgreen LE, Tofts L, et al. Vosoritide therapy in children with achondroplasia aged 3‐59 months: a multinational, randomised, double‐blind, placebo‐controlled, phase 2 trial. Lancet Child Adolesc Health. 2024;8:40–50.
    1. King JA, Vachhrajani S, Drake JM, Rutka JT. Neurosurgical implications of achondroplasia. J Neurosurg Pediatr. 2009;4:297–306.

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