Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS).

Lina TitievskyTilman SchusterRonnie WangMuhammad YounusAndrew PalladinoKabir QuaziMichael P WajnrajchBetina HernandezPamela S BeckerNeal J WeinrebChristina ChambersRoy MansfieldLouise TaylorLi-Jung TsengPaige Kaplan

Published in: Orphanet journal of rare diseases (2022)

The interim data showed no new or emergent safety signals. The overall interim data are consistent with the clinical program experience and known safety and effectiveness profile of taliglucerase alfa.

Keyphrases

electronic health record
randomized controlled trial
replacement therapy
big data
systematic review
quality improvement
data analysis
machine learning
adverse drug
drug induced