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

Weinstein DAR J JacksonE A BrennanM WilliamsJ E DavisonF de BoerTgj DerksC EllertonB FaragherJ GribbenP LabruneK M McKittrickE MurphyK M RossU SteuerwaldC VoillotAjm WoodwardH R Mundy
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
  • end stage renal disease
  • ejection fraction
  • chronic kidney disease
  • study protocol
  • stem cells
  • prognostic factors
  • bone marrow
  • mesenchymal stem cells
  • phase iii