Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in June 2014 on request of the Sponsor.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.
Adeno-associated viral vector serotype 5 containing the human ABCA4 gene
Treatment of Stargardt's disease
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Documents related to this orphan designation evaluation
EU/3/08/609: Public summary of positive opinion for orphan designation of adeno-associated viral vector serotype 5 containing the human ABCA4 gene for the treatment of Stargardt’s disease (PDF/126.34 KB)
First published: 20/03/2009
Last updated: 16/10/2014
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.
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: