P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials
| Main Authors: | , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2023-01-01
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| Series: | Genetics in Medicine Open |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2949774423003771 |
| _version_ | 1797342065121558528 |
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| author | Melissa Wasserstein Roberto Giugliani Simon Jones Robin Lachmann Maurizio Scarpa Monica Kumar Nicole Armstrong Mario Aguiar |
| author_facet | Melissa Wasserstein Roberto Giugliani Simon Jones Robin Lachmann Maurizio Scarpa Monica Kumar Nicole Armstrong Mario Aguiar |
| author_sort | Melissa Wasserstein |
| collection | DOAJ |
| first_indexed | 2024-03-08T10:27:04Z |
| format | Article |
| id | doaj.art-e2b148c6b8fc43aa8e41cbfa23372153 |
| institution | Directory Open Access Journal |
| issn | 2949-7744 |
| language | English |
| last_indexed | 2024-03-08T10:27:04Z |
| publishDate | 2023-01-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Genetics in Medicine Open |
| spelling | doaj.art-e2b148c6b8fc43aa8e41cbfa233721532024-01-27T07:08:00ZengElsevierGenetics in Medicine Open2949-77442023-01-0111100377P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trialsMelissa Wasserstein0Roberto Giugliani1Simon Jones2Robin Lachmann3Maurizio Scarpa4Monica Kumar5Nicole Armstrong6Mario Aguiar7Children’s Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NYHospital de Clínicas de Porto Alegre/Federal University of Rio Grande do Sul, DASA, Casa dos RarosSt Mary's Hospital, Manchester University National Health Service Trust, Manchester, UKCharles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UKRegional Coordinator Centre for Rare Diseases, University Hospital of Udine, Udine, ItalySanofi, Bridgewater, NJSanofi, Cambridge, MASanofi, Cambridge, MAhttp://www.sciencedirect.com/science/article/pii/S2949774423003771 |
| spellingShingle | Melissa Wasserstein Roberto Giugliani Simon Jones Robin Lachmann Maurizio Scarpa Monica Kumar Nicole Armstrong Mario Aguiar P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials Genetics in Medicine Open |
| title | P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials |
| title_full | P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials |
| title_fullStr | P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials |
| title_full_unstemmed | P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials |
| title_short | P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials |
| title_sort | p349 plasma lyso sphingomyelin biomarker for acid sphingomyelinase deficiency correlations with baseline disease and response to olipudase alfa treatment in clinical trials |
| url | http://www.sciencedirect.com/science/article/pii/S2949774423003771 |
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