Why is gene therapy the right treatment option for my child?
Is this treatment part of a clinical trial or an approved therapy?
What are potential treatment risks and uncertainties and their likelihood?
What are the potential benefits of gene therapy for my child?
What are the potential costs to our family?
Will the treatment affect participation in future clinical trials or access to approved treatments?
What preparations must be done for my child to be ready for gene therapy? And are there any pre-infusion medications?
Could you provide me with the additional reading material and information behind your decision so I can understand better?
Is the treatment being offered in Australia different to gene therapy overseas?
What is the timeline like? When do I need to make decisions by?
Procedure
Will you change genes in the cells while the cells are inside the body, or are they taken out first to be treated, then re-introduced to the body?
How is the therapy delivered (injection into blood/vein/spinal fluid/brain)? Why is this the right way for my child? Are there unique risks for this particular delivery method?
How long will the infusion take?
Are there any other potentially complicating factors I should be aware of?
Medical care and recovery
What will the ongoing medical care look like? Are there any post-treatment medications?
How often and for how long will follow-up appointments be necessary?
Are there other risks associated with gene therapy after treatment?
How should I monitor and report side effects if they arise at home?
Should my child continue with the routine immunisation program?
What do you believe are the best support structures in place for children and families going through gene therapy?
If gene therapy changes genes, will my child eventually pass those altered genes onto their children?
