Orphan designation: Adeno-associated viral vector serotype 9 containing the human MECP2 gene, an intron encoding a miRNA generating sequence, and complementary miRNA binding sites Treatment of Rett syndrome, 12/01/2024 Positive
Overview
- Overview
This medicine was designated as an orphan medicine for treatment of the Rett syndrome on 12 January 2024. - Orphan designation does not mean the medicine is available or authorised for use.
- All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
- EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: