A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

• Patisiran (Drug), also known as ONPATTRO, ALN-TTR02: Patisiran-lipid complex injection, for intravenous use

Primary outcomes

• Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline [Baseline, Month 12]: 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.

Sex: All, ages: 18 Years to any

Inclusion Criteria:

* Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
* PND score of I-IIIB at baseline.
* Exposure to commercial patisiran in one of the 3 cohorts:

  * Prospective Cohort: Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran.
  * Mixed cohort: Currently on commercial patisiran therapy for less than 12 months at study enrollment.
  * Retrospective cohort: Exposed to commercial patisiran treatment for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.

Exclusion Criteria:

* New York Heart Association (NYHA) heart failure classification ≥3
* Karnofsky Performance Status (KPS) <60%
* Unstable congestive heart failure (CHF)
* Known primary amyloidosis (AL) or leptomeningeal amyloidosis
* Prior major organ transplant
* Previously received patisiran
* Previous treatment with a TTR silencing therapy

• Medical Director, Study director, Alnylam Pharmaceuticals