Long-term catch-up growth in children with acid sphingomyelinase deficiency treated with olipudase alfa enzyme replacement therapy in the ASCEND-Peds trial

Autor:	Roberto Giugliani, George Diaz, Nathalie Guffon, Simon A. Jones, Margaret McGovern, Eugen Mengel, Maurizio Scarpa, Peter Witters, Abhimanyu Yarramaneni, Nicole Armstrong, Catherine Ortemann-Renon, Yong Kim, Monica Kumar
Rok vydání:	2023
Předmět:	Endocrinology Endocrinology Diabetes and Metabolism Genetics Molecular Biology Biochemistry
Zdroj:	Molecular Genetics and Metabolism. 138:107115
ISSN:	1096-7192
DOI:	10.1016/j.ymgme.2022.107115
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::67eaeef4d90de708f50fd27324eed77b https://doi.org/10.1016/j.ymgme.2022.107115 Zobrazit plný text záznamu Full Text from ScienceDirect