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Table 3 Change in mNIS+7 by components and subgroups at 24 months of patisiran treatment

From: A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

  Change from baseline to Month 24
n Mean (SEM) Median (range)
mNIS+7 component
 Total 26 −6.95 (2.03) −7.00 (−34.63 to 15.38)
 NIS-Weakness 26 1.23 (1.43) 0.00 (−13.50 to 24.38)
 NIS-Reflexes 26 −0.48 (0.53) 0.00 (−6.00 to 7.00)
 QST 26 −7.4 (2.04) −6.0 (−40.0 to 16.0)
 NCS ∑5 26 −0.19 (0.18) −0.25 (−2.00 to 2.50)
 Postural BP 26 −0.10 (0.06) 0.00 (−1.00 to 0.50)
mNIS+7 by TTR tetramer stabilizer use
 Patisiran alone 7 −6.75 (5.24) −8.50 (−28.50 to 15.38)
 Patisiran + TTR tetramer stabilizer 19 −7.03 (2.11) −6.63 (−34.63 to 3.88)
  1. Abbreviations: BP blood pressure, mNIS+7 modified Neuropathy Impairment Score +7, NCS nerve conduction studies, NIS Neuropathy Impairment Score, QST quantitative sensory testing, SEM standard error of the mean, TTR transthyretin