Final results of the ASCEND trial: Continued or sustained improvements in hepatosplenomegaly, respiratory outcomes, and lipid profile after 4 years of olipudase alfa enzyme replacement therapy in adults with acid sphingomyelinase deficiency
MOLECULAR GENETICS AND METABOLISM. Bd. 144. H. 2. 2025
Erscheinungsjahr: 2025
Publikationstyp: Zeitschriftenaufsatz
| Geprüft: | Bibliothek |
Autoren
Wasserstein, Melissa (Autor)
Gallagher, Renata C. (Autor)
Barbato, Antonio (Autor)
Guigliani, Roberto (Autor)
Guelbert, Norberto (Autor)
Hennermann, Julia B. (Autor)
Ikezoe, Takayuki (Autor)
Lachmann, Robin (Autor)
Lidove, Olivier (Autor)
Mabe, Paulina (Autor)
Mengel, Eugen (Autor)
Scarpa, Maurizio (Autor)
Senates, Ebubekir (Autor)
Tchan, Michel (Autor)
Villarrubia, Jesus (Autor)
Thurberg, Beth L. (Autor)
Armstrong, Nicole M. (Autor)
Srivastav, Shruti (Autor)
Wisniewska, Urszula (Autor)
Kumar, Monica (Autor)
Klassifikation
DDC Sachgruppe:
Medizin
