On 1 April 2019, orphan designation (EU/3/19/2149) was granted by the European Commission to Brainvectis, France, for adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene for the treatment of Huntington's disease.
Adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene
Treatment of Huntington’s disease
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Review of designation
|June 2022||The sponsor's address was updated in June 2022.|
Documents related to this orphan designation evaluation
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
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: