ID – 1543
MASSIVE DISEASE BURDEN IN INFANTS WITH GAUCHER DISEASE TYPE 1 AND TYPE 3: LONG-TERM OUTCOMES OF EARLY IMIGLUCERASE THERAPY
AO Dos Santosa, PK Mistryb, JL Carwilec, A Burrowd, J Ganeshe, JS Camelo Jrf, JB Hennermanng, JB Batistac, P Kishnanih, A Tantawyi, MG Perichonc
a Sanofi, Rio de Janeiro, RJ, Brazil
b Yale University School of Medicine, United States
c Sanofi, United States
d University of Arkansas for Medical Sciences and Arkansas Children's Hospital, United States
e Department of Medical Genetics and Genomic Sciences, United States
f Faculdade de Medicina de Ribeirão Preto (FMRP), Universidade de São Paulo (USP), Ribeirão Preto, SP, Brazil
g University Medical Center Mainz, Germany
h Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, United States
i Faculty of Medicine, Ain Shams University, Egypt
Ver más
