| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10833525 | Molecular Genetics and Metabolism | 2012 | 5 Pages |
Abstract
⺠Gaucher disease is the first lysosomal disease for which therapy became available. ⺠Gaucher patients with high disease burden are unlikely to achieve remission. ⺠Attaining reasonably good clinical status is realistic treatment goal for them. ⺠The Authors design the concept of minimal disease activity in Gaucher disease. ⺠Patients with minimal disease activity could be addressed to maintenance therapy.
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Authors
Maja Di Rocco, Generoso Andria, Bruno Bembi, Francesca Carubbi, Fiorina Giona, Gaetano Giuffrida, Silvia Linari, Michelina Sibilio, Vincenzo Spina, Maria Domenica Cappellini,