Update on safety and efficacy results for phase I/II trial of hydroxypropyl betacyclodextrin (HPâCD) administered intravenously in patients with Niemann-Pick disease type C1

Autor:	Reena Sharma, Alan Boyd, Julian Raiman, Orna Staretz-Chacham, Martin Paucar, Sharon Hrynkow, Ronen Spiegel, Caroline A. Hastings
Rok vydání:	2021
Předmět:	medicine.medical_specialty business.industry Endocrinology Diabetes and Metabolism Biochemistry Gastroenterology Endocrinology Phase i ii Internal medicine Genetics medicine In patient business Molecular Biology Niemann-pick disease type
Zdroj:	Molecular Genetics and Metabolism. 132:S90
ISSN:	1096-7192
DOI:	10.1016/j.ymgme.2020.12.215
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::1d003243264b36e50bde2b57931fb6a4 https://doi.org/10.1016/j.ymgme.2020.12.215 Zobrazit plný text záznamu Full Text from ScienceDirect