Author/Editor		Schoser, Benedikt; Roberts, Mark; Byrne, Barry J.; Sitaraman, Sheela; Jiang, Hai; Laforet, Pascal; Toscano, Antonio; Castelli, Jeff; Diaz-Manera, Jordi; Goldman, Mitchel P.; Koritnik, Blaž
Title		Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL)
Type		članek
Vol. and No.		Letnik 20, št. 12
Publication year		2021
Volume		str. 1027-1037
ISSN		1474-4422 - Lancet neurology
Language		eng
Keywords		Pompejeva bolezen otroška nevrologija metabolna miopatija redke bolezni kopičenja glikogena tipa II