在奥利司他α的ASCEND试验中,同型p.Arg610del基因型对成人酸性鞘磷脂酶缺乏症患者的疾病负担和治疗反应的影响

Melissa P. Wasserstein, Monica Kumar, Nicole M. Armstrong, Sefika Uslu Cil, Arya Firoozan, Lisa Underhill, Robin Lachmann
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Impact of homozygous p.Arg610del genotype on disease burden and treatment response in adults with acid sphingomyelinase deficiency in the ASCEND trial of olipudase alfa
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