From: Patients’ view on gene therapy development for lysosomal storage disorders: a qualitative study
Theme | Subtheme | Gaucher | Fabry | MPS III |
---|---|---|---|---|
Outcome of gene therapy | Effectiveness of gene therapy | Gene therapy should be as effective as current therapy | Hope for gene therapy to be more effective than current therapy | Every potential effect gives hope |
Quality of life | ||||
Risks and side effects | Gravity of risks and side effects | Critical attitude | Acceptance | High acceptance |
Duration of side effects | ||||
Burden of gene therapy treatment | Method of gene therapy | Critical attitude | Acceptance | High acceptance |
Practical aspects | ||||
Current situation | Current treatment options | Good | Uncertainty | None |
Beyond-individual aspects | Important | Very important | Extremely important | |
Individual aspects | Differ between group and have different impact | |||
Ethical aspects | Personal and societal aspects | Not disease specific |