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
Orphanet journal of rare diseases（2023）
关键词
Recombinant human acid sphingomyelinase,Dose escalation,Organomegaly,Lung diffusing capacity,Acid sphingomyelinase deficiency,Niemann–Pick type B,Niemann–Pick type A/B
AI 理解论文
溯源树
样例
生成溯源树，研究论文发展脉络
Chat Paper
正在生成论文摘要