Login / Signup

Short and long-term acceptability and efficacy of extended-release cornstarch in the hepatic glycogen storage diseases: results from the Glyde study.

Weinstein DAJackson RjBrennan EaWilliams MDavison Jede Boer FDerks TgjEllerton CFaragher BGribben JLabrune PMcKittrick KmMurphy ERoss KmSteuerwald UVoillot CWoodward AjmMundy Hr
Published in: Orphanet journal of rare diseases (2024)
The Glyde study is the first multi-centre international trial demonstrating the efficacy and tolerance of Glycosade in a large cohort of hepatic GSD patients across a diverse international population. The ability to use fewer doses of therapy per day and avoidance of overnight therapy may improve compliance, safety, and quality of life without sacrificing metabolic control.
Keyphrases
  • newly diagnosed
  • ejection fraction
  • clinical trial
  • randomized controlled trial
  • mesenchymal stem cells
  • phase ii