On 10 August 2015, orphan designation (EU/3/15/1539) was granted by the European Commission to Audentes Therapeutics UK Limited, United Kingdom, for adeno-associated viral vector serotype 8 containing the human MTM1 gene for the treatment of X-linked myotubular myopathy.
The sponsorship was transferred to Audentes Therapeutics Netherlands B.V., The Netherlands, in April 2019.
Adeno-associated viral vector serotype 8 containing the human MTM1 gene
|Disease / condition||
Treatment of X-linked myotubular myopathy
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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;