Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial

Line Borgwardt,Nathalie Guffon,Yasmina Amraoui,Christine I. Dali,Linda De Meirleir,Mercedes Gil-Campos,Bénédicte Héron,Silvia Geraci,Diego Ardigò,Federica Cattaneo,Jens Fogh,J. M. Hannerieke Van den Hout,Michael Beck,Simon A. Jones,Anna Tylki-Szymańska,Ulla Haugsted,Allan M. Lund

Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial

2018

Line Borgwardt
Nathalie Guffon
Yasmina Amraoui
Christine I. Dali
Linda De Meirleir
Mercedes Gil-Campos
Bénédicte Héron
Silvia Geraci
Diego Ardigò
Federica Cattaneo
Jens Fogh
J. M. Hannerieke Van den Hout
Michael Beck
Simon A. Jones
Anna Tylki-Szymańska
Ulla Haugsted
Allan M. Lund

Introduction This phase III, double-blind, randomised, placebo-controlled trial (and extension phase) was designed to assess the efficacy and safety of velmanase alfa (VA) in alpha-mannosidosis (AM) patients.

Keywords:

Alpha-mannosidosis
Endocrinology
Biology
Diabetes mellitus
Velmanase alfa
Placebo-controlled study
Internal medicine
Post-hoc analysis
Placebo
double blind
Confidence interval
phase analysis

Correction
Source
Cite
Save
Machine Reading By IdeaReader

References

Citations