奥利肽酶α酶替代疗法可逆转酸性鞘磷脂酶缺乏症成人的间质性肺病:ASCEND试验的长期肺部疗效

Melissa P. Wasserstein, F. Bonella, Roberto Giugliani, Robin Lachmann, Imre Noth, Maurizio Scarpa, Wim A. Wuyts, Mario Aguiar, Nicole Armstrong, Monica Kumar, Kenneth I Berger
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Olipudase alfa enzyme replacement therapy reverses interstitial lung disease in adults with acid sphingomyelinase deficiency: Long-term pulmonary outcomes of the ASCEND trial
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