A retrospective survey of patients with hereditary transthyretin-mediated (hattr) amyloidosis treated with patisiran in real-world clinical practice in belgium

HIGHLIGHTS

  • who: Jan L. De Bleecker from the Department of Neurology, University Hospital Ghent, Ghent, Belgium have published the article: A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium, in the Journal: (JOURNAL) of 01/07/2018
  • what: The study shows that the efficacy of patisiran, as reported in the initial pivotal clinical trials , is confirmed in real-world clinical practice over a median period of 663 days since treatment initiation. This study has several limitations, such as a small population sample preventing analytic . . .

     

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