To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
Study Type
OBSERVATIONAL
Enrollment
67
Patisiran-lipid complex injection, for intravenous use
Clinical Trial Site
Los Angeles, California, United States
Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline
PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.
Time frame: Baseline, Month 12
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Chicago, Illinois, United States
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...and 17 more locations