Sanfilippo C: TBA
University of Barcelona. Department of Genetic
Dr. Daniel Grinberg dgrinberg@ub.edu
Dr. Lluïsa Vilageliu lvilageliu@ub.edu
Gene Therapy Type A
University of Barcelona
Dr. Fatima Bosch fatima.bosch@uab.es
This week in the meeting of The Committee for Orphan Medicinal Products (COMP), has been presented to the European Medicines Agency (EMEA) the drug “Adeno-associated virus vector serotype 9 expressing human sulfamidase for treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome), D. Esteve Labs, S.A. – EMA/OD/171/10)