P1483: LONG-TERM IMPACT OF OLIPUDASE ALFA ENZYME REPLACEMENT THERAPY ON SPLEEN VOLUME AND HEMATOLOGIC MANIFESTATIONS IN CHILDREN AND ADULTS WITH CHRONIC ACID SPHINGOMYELINASE DEFICIENCY
Jesus Villarrubia,
Jaya Ganesh,
Roberto Giugliani,
Nathalie Guffon,
Eugen Mengel,
Maurizio Scarpa,
Melissa Wasserstein,
Nicole Armstrong,
Gasparic Maja,
Yong Kim,
Holly Wong,
Abhimanyu Yarramaneni
Affiliations
Jesus Villarrubia
1 Ramón y Cajal Hospital, Hematology, Madrid, Spain
Jaya Ganesh
2 Icahn School of Medicine at Mount Sinai, Genetics and Genomic Sciences, New York, NY, United States
Roberto Giugliani
3 Genetics UFRGS, Medical Genetics Service HCPA, DASA and Casa dos Raros, Porto Alegre, Brazil
Nathalie Guffon
4 Hôpital Femme Mère Enfant- Hospices Civils de Lyon, Centre de Référence des Maladies Héréditaires du Métabolisme, Lyon, France
Eugen Mengel
5 Institute of Clinical Science for Lysosomal Storage Disorders, SphinCS GmbH, Hochheim, Germany
Maurizio Scarpa
6 University Hospital of Udine, Regional Coordinator Centre for Rare Diseases, Udine, Italy
Melissa Wasserstein
7 Albert Einstein College of Medicine, Children’s Hospital at Montefiore, Bronx, NY, United States
