Increased intervals in enzyme replacement therapy for stable type 1 Gaucher disease: A non-inferiority sequential trial emulation.
Beydon M, Stirnemann J, Yousfi K, Zebiche S, Hamroun D, Brassier A, Pichard S, Swiader L, de Villemeur TB, Héron B, Dalbies F, Cador B, Guemann AS, Gaches F, Hivert B, Leguy-Seguin V, Masseau A, Pers YM, Pettazzoni M, Bekri S, Caillaud C, Guillou EL, Szymanowski M, Astudillo L, Mauhin W, Nadjar Y, Serratrice C, Berger MG, Camou F, Belmatoug N, Nguyen Y; French Evaluation of Gaucher Disease Treatment Committee.
Beydon M, et al. Among authors: camou f.
J Intern Med. 2026 May;299(5):604-614. doi: 10.1111/joim.70079. Epub 2026 Feb 28.
J Intern Med. 2026.
PMID: 41761869
Free PMC article.
Clinical Trial.