EU/3/14/1282
Table of contents
About
On 4 July 2014, orphan designation (EU/3/14/1282) was granted by the European Commission to Fondazione Telethon, Italy, for mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene for the treatment of Usher syndrome.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Key facts
Active substance |
Mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene
|
Disease / condition |
Treatment of Usher syndrome
|
Date of first decision |
04/07/2014
|
Outcome |
Positive
|
EU designation number |
EU/3/14/1282
|
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Sponsor's contact details
Fondazione Telethon
Via Varese 16
00185 Roma
Italy
Tel. +39 06 44 01 51
Fax +39 06 44 01 55 21
E-mail: info@telethon.it
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.