Priority 12 from the Liver Glycogen Storage Disease (International) PSP
| UNCERTAINTY: What are the risks and benefits of gene therapy for patients with liver Glycogen Storage Disease? (JLA PSP Priority 12) | |
|---|---|
| Overall ranking | 12 |
| JLA question ID | 0090/12 |
| Explanatory note |
A gene therapy trial for GSD Ia is currently being performed and gene therapy is being developped for other types of liver GSD. This question stresses that it is important to evaluate short-term and long-term risks and benefits of this new treatment option. |
| Evidence |
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 | What side effects will gene therapy have? ~ Long-term studies - stem cells / gene therapy |
| 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 |