Page 329 - Programme book for The International Liver Congress 2015, EASL ILC 2015

Genetic and Pediatric Liver Diseases

P1211 EFFICACY AND SAFETY OF SEBELIPASE ALFA IN
CHILDREN AND ADULTS WITH LYSOSOMAL ACID LIPASE
DEFICIENCY: RESULTS OF A PHASE 3 TRIAL
Sandra Rojas-Caro, Ivo Baric, Carmen Camarena Grande,
Mahmut Coker, Patrick Deegan, Maja DiRocco, Fatih S. Ezgu,
Francois Feillet, Vera Malinova, Eugen Mengel, Elaine Murphy,
Jose Pastor Rosado,Yusof Rahman, Maurizio Scarpa, Karl O. Schwab,
Vratislav Smolka, Joanna Taybert, Vassili Valayannopoulos,Yijun Yang,
Anthony Quinn*, The United States

P1212 IMPACT OF SEBELIPASE ALFA ON SURVIVAL AND LIVER
FUNCTION IN INFANTS WITH RAPIDLY PROGRESSIVE
LYSOSOMAL ACID LIPASE DEFICIENCY
Simon Jones, Dominique Plantaz, Roshni Vara, John J. Gargus,
Joanne Hughes, Sandra Rojas-Caro, Anthony G. Quinn*,
Vassili Valayannopoulos, The United States

P1213 ADENO-ASSOCIATED VIRUS VECTOR-MEDIATED LIVER
GENE THERAPY FOR CRIGLER-NAJJAR SYNDROME
Piter Bosma, Philippe Labrune, Nicola Brunetti-Pierri, ePOSTERS
Lorenzo D’Antiga, Michael Ott, Marylene Beinat, Flavio Ronchi,
Fulvio Mavilio, Ulrich Beuers, Andres Muro, Ulrich Baumann,
Federico Mingozzi*, France

P1214 DIET HIGHLY INFLUENCES THE DEVELOPMENT OF
HEPATOCELLULAR ADENOMAS AND CARCINOMAS IN
MICE WITH GLYCOGEN STORAGE DISEASE TYPE IA
Julie Clar, Margaux Raffin, Sophie Gaillard, Olivier Beuf,
Gilles Mithieux, Fabienne Rajas*, France

P1215 INVITRO EVALUATION OF ANTISENSE DRUGS IN
METABOLIC LIVER DISEASE-SPECIFIC HEPATOCYTE-
LIKE CELLS USING IPS-CELL TECHNOLOGY
Vanessa Sauer*, Christoph Niemietz, Gursimran Chandhok,
Andree Zibert, Hartmut H.-J. Schmidt, Germany

P1216 ADVANCED LIVER DISEASE CORRELATES WITH LOW
SERUM LYSOSOMAL ACID LIPASE ACTIVITY
Eyal Shteyer, Rivka Villenchik, Mahmud Mahamid, Nidaa Nator,
Rifaat Safadi*, Israel

Vienna, Austria • April 22–26, 2015 329