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ONPATTRO® (patisiran) improved polyneuropathy symptoms related to hATTR amyloidosis

Patients receiving ONPATTRO showed improvement in nerve function compared to those who received placebo

Significant improvements in nerve function as measured by:

  • Strength in hands and feet, arms and legs
  • Blood pressure upon standing
  • Reflexes
  • Response to temperature/vibration
 

More than half of patients (56%) treated with ONPATTRO showed improvement in nerve function from their starting point in the study, compared to placebo (4%).

Change in nerve function from baseline at 18 monthsa

Change in nerve function from baseline at 18 months

aNerve function was measured using a 304-point scale called the modified Neuropathy Impairment Score (mNIS) +7.

Patients receiving ONPATTRO showed improvement in quality of life compared to those who received placebo

Significant improvements in quality of life, compared to placebo, as measured byb:

  • Symptoms of neuropathy (numbness, tingling, pain, weakness, etc.)
  • Digestive health
  • Ability to perform everyday activities (dressing, bathing, walking, etc.)
  • Steadiness on feet

Change in quality of life from baseline at 18 monthsb

Change in quality of life from baseline at 18 months
bChange in quality of life was measured using a questionnaire (score range -4 to 136) called the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) that evaluated the effect of the disease on patients’ daily life.

Patients treated with ONPATTRO also showed improvements in other measures

At 18 months, when compared to placebo, patients experienced:

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Decrease in symptoms
such as diarrhea, constipation, dizziness, difficulty urinating, and vomiting

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Better nutritional health
ONPATTRO-treated patients maintained a healthier weight

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Increase in walking speed
ONPATTRO-treated patients could walk faster

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Decrease in pain

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Decreased feelings of anxiety and depression