Priority 11 from the Liver Glycogen Storage Disease (International) PSP

UNCERTAINTY: How to prevent and/or treat muscle problems in patients with liver Glycogen Storage Disease?  (JLA PSP Priority 11)
Overall ranking 11
JLA question ID 0090/11
Explanatory note

Muscle problems are an important complication of liver GSD that also have a muscle phenotype and are associated with possible decrease in quality of life. This question aims to prevent and/or treat these muscle complications. 


For details of the evidence checked, please see the spreadsheet held on the JLA website.

Health Research Classification System category Metabolic and endocrine 
Extra information provided by this PSP
Original uncertainty examples Myopathy for GSD Type III ~ Skeletal muscle involvement ~ Is there a solution to treat my sick muscles?  
Submitted by Patient, carer and healthcare professionals 
PSP information
PSP unique ID 0090
PSP name International Glycogen Storage Disease PSP
Total number of uncertainties identified by this PSP 72  (To see a full list of all uncertainties identified, please see the detailed spreadsheet held on the JLA website)
Date of priority setting workshop 24 May 2019