1. McGovern MM, Dionisi-Vici C, Giugliani R, et al. Consensus recommendation for a diagnostic guideline for acid sphingomyelinase deficiency. Genet Med. 2017;19(9):967–74.
2. Wasserstein M, Dionisi-Vici C, Giugliani R, et al. Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD). Mol Genet Metab. 2019;126(2):98–105.
3. Sanofi. Xenpozyme®(olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency [media release]. 28 Mar 2022. http://www.sanofi.com.
4. Sanofi. Olipudase alfa (XENPOZYME®) IV infusion: Japanese prescribing information. 2022. https://www.pmda.go.jp/PmdaSearch/iyakuDetail/780069_39594A9D1022_1_01. Accessed 31 Mar 2022
5. Sanofi. CHMP recommends approval of Xenpozyme® (olipudase alfa), the first and only treatment for ASMD [media release]. 20 May 2022. https://www.sanofi.com.