EU/3/18/2007

Table of contents

About

On 16 April 2018, orphan designation (EU/3/18/2007) was granted by the European Commission to Dr Philippe Moullier, France, for adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene (also known as AAV2/8-LSPhGAA) for the treatment of glycogen storage disease type II (Pompe's disease).

Key facts

Active substance
Adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene
Disease / condition
Treatment of glycogen storage disease type II (Pompe's disease)
Date of decision
16/04/2018
Outcome
Positive
Orphan decision number
EU/3/18/2007

Sponsor's contact details

Dr Philippe Moullier
1 rue du Roi Albert
44000 Nantes
France
Tel. +33 617 13 86 62
E-mail: pmoullier@outlook.com

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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