The journey
The whole treatment, in order, from the first conversation to long-term follow-up. One step at a time.
Eleven stops. Tap next, or jump to the part you need.
Written from published sources
This page is written from published guidance and research by regulators, the NHS and peer-reviewed journals. Every source is listed at the bottom of the page so you can check it yourself.
It has not been reviewed by a clinician. It is general information, not advice about your own care. Always check anything that matters with your own medical team.
This page shows the shape and order of the pathway. It does not describe what any step will be like for a particular person. That varies enormously, and it is a conversation for your own team. Where something has not yet been written from a published source, that is said rather than guessed.
Step 1 of 11
A dotted path leading from one person to another, showing a referral being made to a treatment centre.
Getting to the right team
This is the first conversation. Someone refers you to a specialist centre that can talk about gene therapy as an option.
Why is gene therapy being offered now?
Who decides whether we go ahead, and when?
How long do we have to make this decision?
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