Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial
ORPHANET JOURNAL OF RARE DISEASES. Bd. 18. H. 1. 2023
Erscheinungsjahr: 2023
Publikationstyp: Zeitschriftenaufsatz
| Geprüft: | Bibliothek |
Autoren
Wasserstein, Melissa P. (Autor)
Lachmann, Robin (Autor)
Hollak, Carla (Autor)
Barbato, Antonio (Autor)
Gallagher, Renata C. (Autor)
Giugliani, Roberto (Autor)
Guelbert, Norberto Bernardo (Autor)
Hennermann, Julia B. (Autor)
Ikezoe, Takayuki (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)
Yarramaneni, Abhimanyu (Autor)
Armstrong, Nicole M. (Autor)
Kim, Yong (Autor)
Kumar, Monica (Autor)
Klassifikation
DDC Sachgruppe:
Medizin
