Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months

Melissa P. Wasserstein, George A. Diaz, Robin H. Lachmann, Marie Hélène Jouvin, Indrani Nandy, Allena J. Ji, Ana Cristina Puga

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79 Scopus citations

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Medicine & Life Sciences