DNL310 normalizes primary storage substrates and biomarkers of lysosomal dysfunction in neuronopathic MPS II: 2-year interim analysis of a phase 1/2 study

Autor:	Akhil Bhalla, Khishig Tsogtbaatar, Ritesh Ravi, Meng Fang, Sonnet S. Davis, Imanol Zubizarreta, Ajith Karunakara, Yuda Zhu, Elizabeth Sinclair, Dominique Jacquemet-Engelhart, Shyeilla V. Dhuria, Tony Hung, Matthew Troyer, Peter Chin, Jeff M. Harris, Anna Bakardjiev
Rok vydání:	2023
Předmět:	Endocrinology Endocrinology Diabetes and Metabolism Genetics Molecular Biology Biochemistry
Zdroj:	Molecular Genetics and Metabolism. 138:107031
ISSN:	1096-7192
DOI:	10.1016/j.ymgme.2022.107031
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::adf4018aa398d509df431f80b0f78801 https://doi.org/10.1016/j.ymgme.2022.107031 Zobrazit plný text záznamu Full Text from ScienceDirect