Continued improvement in pulmonary, visceral, biomarker and growth outcomes in children with chronic acid sphingomyelinase deficiency treated with olipudase alfa enzyme replacement therapy: 2-year results of ASCEND-Peds

Autor:	George A. Diaz, Roberto Giugliani, Nathalie Guffon, Eugen Mengel, Maurizio Scarpa, Peter Witters, Abhimanyu Yarramaneni, Jing Li, Andreea M. Rawlings, Yong Kim, Catherine Ortemann-Renon, Monica Kumar
Rok vydání:	2022
Předmět:	Endocrinology Endocrinology Diabetes and Metabolism Genetics Molecular Biology Biochemistry
Zdroj:	Molecular Genetics and Metabolism. 135:S37
ISSN:	1096-7192
DOI:	10.1016/j.ymgme.2021.11.082
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::c5e6d6630dbb69f50334434e94a8ed9b https://doi.org/10.1016/j.ymgme.2021.11.082 Zobrazit plný text záznamu Full Text from ScienceDirect