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Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in June 2014 on request of the Sponsor.

On 6 February 2009, orphan designation (EU/3/08/609) was granted by the European Commission to Fondazione Telethon, Italy, for adeno-associated viral vector serotype 5 containing the human ABCA4 gene for the treatment of Stargardt's disease.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

Key facts

Active substance
Adeno-associated viral vector serotype 5 containing the human ABCA4 gene
Disease / condition
Treatment of Stargardt's disease
Date of first decision
EU designation number

Sponsor's contact details

Fondazione Telethon
Via dei Magazzini Generali 18/20
00154 Roma
Tel. +39 06 44 01 51
Fax +39 06 44 20 20 32

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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