Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use

Author:

Syed Yahiya Y.

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),General Medicine

Reference33 articles.

1. Slotte JP. Biological functions of sphingomyelins. Prog Lipid Res. 2013;52(4):424–37.

2. Schuchman EH, Desnick RJ. Types A and B Niemann–Pick disease. Mol Genet Metab. 2017;120(1–2):27–33.

3. 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.

4. McGovern MM, Wasserstein MP, Bembi B, et al. Prospective study of the natural history of chronic acid sphingomyelinase deficiency in children and adults: eleven years of observation. Orphanet J Rare Dis. 2021;16(212):1–14.

5. McGovern MM, Avetisyan R, Sanson BJ, et al. Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD). Orphanet J Rare Dis. 2017;12(41):1–13.

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