EU/3/14/1282

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.

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