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1 Ergebnisse
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Continued improvement in disease manifestations of acid sph..:
Giugliani, Roberto
;
Guelbert, Norberto
;
Hennermann, Julia
...
qt3tx7861m. , 2023
Link:
https://escholarship.org/uc/item/3tx7861m
RT Journal T1
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
UL https://suche.suub.uni-bremen.de/peid=base-ftcdlib:oai:escholarship.org:ark:_13030_qt3tx7861m&Exemplar=1&LAN=DE A1 Giugliani, Roberto A1 Guelbert, Norberto A1 Hennermann, Julia A1 Ikezoe, Takayuki A1 Lidove, Olivier A1 Mabe, Paulina A1 Mengel, Eugen A1 Scarpa, Maurizio A1 Senates, Ebubekir A1 Tchan, Michel A1 Villarrubia, Jesus A1 Thurberg, Beth A1 Yarramaneni, Abhimanyu A1 Armstrong, Nicole A1 Kim, Yong A1 Kumar, Monica A1 Wasserstein, Melissa A1 Lachmann, Robin A1 Hollak, Carla A1 Barbato, Antonio A1 Gallagher, Renata PB eScholarship, University of California YR 2023 K1 Acid sphingomyelinase deficiency K1 Dose escalation K1 Lung diffusing capacity K1 Niemann–Pick type A/B K1 Niemann–Pick type B K1 Organomegaly K1 Recombinant human acid sphingomyelinase K1 Adult K1 Humans K1 Sphingomyelin Phosphodiesterase K1 Niemann-Pick Disease K1 Type A K1 Niemann-Pick Diseases K1 Recombinant Proteins JF qt3tx7861m LK http://dx.doi.org/https://escholarship.org/uc/item/3tx7861m DO https://escholarship.org/uc/item/3tx7861m SF ELIB - SuUB Bremen
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