P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials
Melissa Wasserstein,
Roberto Giugliani,
Simon Jones,
Robin Lachmann,
Maurizio Scarpa,
Monica Kumar,
Nicole Armstrong,
Mario Aguiar
Affiliations
Melissa Wasserstein
Children’s Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY
Roberto Giugliani
Hospital de Clínicas de Porto Alegre/Federal University of Rio Grande do Sul, DASA, Casa dos Raros
Simon Jones
St Mary's Hospital, Manchester University National Health Service Trust, Manchester, UK
Robin Lachmann
Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK
Maurizio Scarpa
Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, Udine, Italy
