114. Pharmacological chaperone therapy for Gaucher disease: Mechanism of action, a survey of responsive mutations and phase I clinical trial results

Autor:	C.W. Pine, Frank Insinga, Paul M. Fernhoff, Paige Kaplan, Gregory A. Grabowski, Neal J. Weinreb, David J. Lockhart, John J. Flanagan, Do Hung, David Palling, Brandon Wustman, Gregory M. Pastores, Brian Ranes
Rok vydání:	2008
Předmět:	Pathology medicine.medical_specialty business.industry Endocrinology Diabetes and Metabolism Phases of clinical research Disease Bioinformatics Biochemistry Pharmacological chaperone Endocrinology Mechanism of action Genetics medicine medicine.symptom business Molecular Biology medicine.drug
Zdroj:	Molecular Genetics and Metabolism. 93:44
ISSN:	1096-7192
DOI:	10.1016/j.ymgme.2007.10.126
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::d7a8efa84a8c423d45f257bd6d685912 https://doi.org/10.1016/j.ymgme.2007.10.126 Zobrazit plný text záznamu Full Text from ScienceDirect