Genetic conditions are caused by inherited changes in a person’s genetic material (DNA, chromosomes).
Some genetic conditions have effective treatments available. Depending on the condition, treatments may include one of the following approaches:
- dietary management
- stem cell transplantation
- enzyme replacement therapy
- drug therapies
- surgical procedures
- gene therapy
Gene therapy is a specific treatment technique that aims to change a person’s genetic material (DNA or RNA) to treat or cure disease.
Voretigene neparvovec (Luxturna)
Voretigene neparvovec (Luxturna) is a gene replacement therapy for people with inherited retinal dystrophy. It is approved by Health Canada for treatment of adults and children, over the age of four, who have mutations in both copies of the RPE65 gene and who meet other eligibility criteria.
This treatment requires specialized expertise and equipment that is only available at two Ontario hospitals:
- The Hospital for Sick Children (for children and young adults)
- Sunnybrook Health Sciences Centre (for adults)
Talk to your health care team to see if you may be eligible for voretigene neparvovec gene therapy. If this is an appropriate therapy for you, your health care team will apply for provincial funding for the treatment.