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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
Autores principales: | Villarrubia, J., Wasserstein, M., Barbato, A., Gallagher, R. C., Giugliani, R., Guelbert, N. B., Hennermann, J. B., Hollak, C., Ikezoe, T., Lachmann, R., Lidove, O., Mabe, P., Mengel, E., Scarpa, M., Senates, E., Tchan, M., Thurberg, B. L., Yarramaneni, A., Rawlings, A. M., Kim, Y., Kumar, M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Lippincott Williams & Wilkins
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9431580/ http://dx.doi.org/10.1097/01.HS9.0000852080.43506.a5 |
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