Information for the public

Eladocagene exuparvovec (Upstaza) is available on the NHS. It is a possible treatment for aromatic L‑amino acid decarboxylase (AADC) deficiency in people 18 months and over if they have a clinical, molecular and genetically confirmed diagnosis of AADC deficiency with a severe phenotype.

Is this treatment right for me or my child?

Your healthcare professionals should give you clear information, talk with you about your or your child’s options, and listen carefully to your views and concerns. Your family can be involved too, if you wish. Read more about making decisions about your care.

Questions to think about

  • How well does it work compared with other treatments?
  • What are the risks or side effects? How likely are they?
  • How will the treatment affect my or my child’s day-to-day life?
  • What happens if the treatment does not work?
  • What happens if I do not want to have treatment or I do not want my child to have it? Are there other treatments available?

Information and support

These organisations can give you advice and support:

You can also get support from your local Healthwatch.

NICE is not responsible for the quality or accuracy of any information or advice provided by these organisations.

ISBN: 978-1-4731-5147-5

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