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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

Melissa P. Wasserstein,Robin Lachmann,Carla Hollak,Antonio Barbato,Renata C. Gallagher,Roberto Giugliani,Norberto Bernardo Guelbert,Julia B. Hennermann,Takayuki Ikezoe,Olivier Lidove,Paulina Mabe,Eugen Mengel,Maurizio Scarpa,Ebubekir Senates,Michel Tchan,Jesus Villarrubia,Beth L. Thurberg,Abhimanyu Yarramaneni,Nicole M. Armstrong,Yong Kim,Monica Kumar

Orphanet Journal of Rare Diseases（2023）

Cited 0|Views23
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Key words
Recombinant human acid sphingomyelinase,Dose escalation,Organomegaly,Lung diffusing capacity,Acid sphingomyelinase deficiency,Niemann–Pick type B,Niemann–Pick type A/B
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