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Glycogen storage disease type III: a mixed-methods study to assess the burden of disease.

Ayla EvinsJill MayhewTricia CimmsJulie WhyteKathy VongElizabeth HribalChristopher J EvansAndrew Grimm
Published in: Therapeutic advances in endocrinology and metabolism (2024)
Individuals with GSD III experience a broad spectrum of symptoms and disease impacts. There is an unmet need for therapies that improve metabolic control, reduce the burden of dietary management, reduce fatigue and liver problems, and improve muscle strength and function.
Keyphrases
  • type iii
  • mental health
  • sleep quality
  • risk factors
  • depressive symptoms