Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Exclusion Criteria

• •Low Retinol level at screen
• •Karnofsky performance status ≤50
• •Poor Renal function
• •Known type 1 or type 2 diabetes mellitus
• •Other causes of sensorimotor or autonomic neuropathy (for example, autoimmune disease)
• •If previously treated with Vyndaqel®, will need to have discontinued treatment for 2 weeks prior to Study Day 1. If previously treated with Diflunisal, will need to have discontinued treatment for 3 days prior to Study Day 1
• •Previous treatment with any oligonucleotide or siRNA within 12 months of screening
• •Prior liver transplant or anticipated liver transplant within 1 year of screening
• •New York Heart Association (NYHA) functional classification of ≥3
• •Acute Coronary Syndrome or major surgery within 3 months of screening
• •Known Primary or Leptomeningeal Amyloidosis
• •Anticipated survival less than 2 years
• •Any other conditions in the opinion of the investigator which interfere with the participant participating in or completing the study