PB2314: OLIPUDASE ALFA FOR ADULTS WITH ACID SPHINGOMYELINASE DEFICIENCY: IMPROVEMENTS IN CROSSOVER PLACEBO PATIENTS AND FURTHER IMPROVEMENTS IN ORIGINAL OLIPUDASE ALFA PATIENTS AFTER 2 YEARS IN ASCEND TRIAL

• J. Villarrubia,
• M. Wasserstein,
• A. Barbato,
• R. C. Gallagher,
• R. Giugliani,
• N. B. Guelbert,
• J. B. Hennermann,
• C. Hollak,
• T. Ikezoe,
• R. Lachmann,
• O. Lidove,
• P. Mabe,
• E. Mengel,
• M. Scarpa,
• E. Senates,
• M. Tchan,
• B. L. Thurberg,
• A. Yarramaneni,
• A. M. Rawlings,
• Y. Kim,
• M. Kumar

Affiliations

J. Villarrubia

1 Hematology, Hospital Universitario Ramón y Cajal, Madrid, Spain

M. Wasserstein

2 Children’s Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY, United States of America

A. Barbato

3 Clinical Medicine and Surgery, Federico II University Hospital, Naples, Italy

R. C. Gallagher

4 Pediatrics, University of California, San Francisco, CA, United States of America

R. Giugliani

5 Med Genet Serv & DR BRASIL Research Grp, HCPA, Dept Genetics, UFRGS & INAGEMP, Porto Alegre, Brazil

N. B. Guelbert

6 Reina Fabiola University Clinic, Córdoba, Argentina

J. B. Hennermann

7 Pediatric and Adolescent Medicine, Villa Metabolica, University Medical Center, Mainz, Germany

C. Hollak

8 Endocrinology and Metabolism, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands

T. Ikezoe

9 Hematology, Fukushima Medical University, Fukushima, Japan

R. Lachmann

10 Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, United Kingdom

O. Lidove

11 La Croix St Simon Hospital, Paris, France

P. Mabe

12 Clinica Santa Maria, Santiago, Chile

E. Mengel

13 Clinical Science for LSD, SpinCS, Hochheim, Germany

M. Scarpa

14 Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, Udine, Italy

E. Senates

15 Istanbul Medeniyet University, Istanbul, Turkey

M. Tchan

16 Genetic Medicine, Westmead Hospital, Sydney, Australia

B. L. Thurberg

17 Sanofi, Cambridge, MA

A. Yarramaneni

18 Sanofi, Bridgewater, NJ, United States of America

A. M. Rawlings

18 Sanofi, Bridgewater, NJ, United States of America

Y. Kim

19 Sanofi, Paris, France

M. Kumar

18 Sanofi, Bridgewater, NJ, United States of America