In an 18-month clinical study, AMVUTTRA-treated patients showed significant improvement in nerve function and quality of life at 9 months and continued to improve throughout the study, compared with those who received placebo in a similar study.
Nerve function
Quality of Life/p>
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At 9 months, AMVUTTRA significantly improved nerve function while patients who received placebo got worse.
At 18 months, 48% of AMVUTTRA-treated patients regained some nerve function from the start of treatment compared with 4% of those who received placebo.
- For the patients who received AMVUTTRA and did not regain some nerve function, progression of their neuropathy was slowed compared with those who received placebo
Nerve function was assessed using a scale called mNIS+7 that measured strength and sensation in the hands, feet, arms, and legs; reflexes; and blood pressure upon standing.a
aHigher scores indicate more severe disease (total score ranges from 0 to 304).
At 9 months, AMVUTTRA significantly improved quality of life while patients who received placebo got worse.
At 18 months, 57% of AMVUTTRA-treated patients reported better quality of life from the start of treatment compared with 10% of those who received placebo.
Quality of life was evaluated by the Norfolk QoL-DN questionnaire that asked patients about the severity of their polyneuropathy symptoms, how often they experienced them, and what impact they felt they had on their daily lives.b
bHigher scores indicate more severe impact of polyneuropathy symptoms on daily life (total score ranges from -4 to 136).
Ask your doctor, “Is AMVUTTRA right for me?”
The effects of AMVUTTRA on other aspects of the disease, including walking speed, nutritional health, and ability to perform activities of daily living, were also evaluated at 18 months and compared with the effects of placebo in a similar study.
AMVUTTRA-treated patients maintained a better walking speed, compared with patients who received placebo
- Evaluated using the 10-meter walk test, a stopwatch-timed measure of a patient’s walking speed over 10 meters
AMVUTTRA-treated patients experienced improvement in nutritional health, compared with patients who received placebo
- Evaluated using modified body mass index, an assessment of height, weight, and the balance of fluids in the body
- In patients with the polyneuropathy caused by the disease, gastrointestinal distress can lead to a decline in nutritional health
AMVUTTRA-treated patients were better able to perform common daily activities, compared with patients who received placebo
- Assessed using a 24-item questionnaire called R-ODS, in which patients rated their ability to complete tasks at the beginning of the study and at the end of the study
- Common daily activities included showering, brushing teeth, dressing upper body, going shopping, doing dishes, and walking up a flight of stairs
