At a glance: the largest Niemann-Pick type C1 cohort with 602
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Methods and compositions for treating a proprotein convertase subtilisin kexin (PCSK9) gene-associated disorder Patent Grant Fitzgerald December 1, 2 [Alnylam Pharmaceuticals, Inc.]
Clinical disease characteristics of patients with Niemann-Pick Disease Type C: findings from the International Niemann-Pick Disease Registry (INPDR). - Abstract - Europe PMC
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PDF) At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years
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