EuropeResearch and clinical trials
In France, gene therapy delivered to the brain improved movement in 6 children with AADC deficiency
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
In France, six patients with AADC deficiency received gene therapy delivered to both sides of the brain. Movement skills improved in all patients. Eye-rolling crises (oculogyric crises) decreased. No serious side effects were seen. In the early period there were temporary involuntary movements, which later resolved.
What does it mean for patients and families?
This is a very small group of patients. This treatment is given only at specialist centers; access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
Scientific paper: Gene Therapy for Amino Acid Decarboxylase Deficiency: Clinical and Imaging Outcomes in a French Cohort. Movement Disorders, 2026. DOI: 10.1002/mds.70422 · PubMed 42389831
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Aromatic L-amino acid decarboxylase (AADC) deficiency
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.