Upstaza™

First gene therapy recommended for AADC

23 March 2023 | By Catherine Eckford (European Pharmaceutical Review)

NICE's final draft guidance has recommended the first and only gene therapy for children with aromatic L-amino acid decarboxylase (AADC) deficiency.

news

EMA releases human medicines highlights 2022 report

17 February 2023 | By Catherine Eckford (European Pharmaceutical Review)

Medicines which represented significant progress in their therapeutic areas have been listed in the European Medicines Agency's Human Medicines Highlights 2022 report.

news

MHRA approves first gene therapy for infusion into the brain

18 November 2022 | By Catherine Eckford (European Pharmaceutical Review)

Upstaza™, approved for UK patients 18 months and over, is the first gene replacement therapy infused directly into the brain for the treatment of AADC deficiency.

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Recommended

Upstaza™

First gene therapy recommended for AADC

EMA releases human medicines highlights 2022 report

MHRA approves first gene therapy for infusion into the brain