hATTR amyloidosis is an inherited, progressive disease caused by a genetic mutation that results in the buildup of misfolded transthyretin (TTR) protein. This results in the formation of amyloid deposits in the heart, nerves, and gastrointestinal tract. hATTR amyloidosis affects approximately 50,000 people worldwide. Treatment options for people with this disease are limited and many people remain undiagnosed or misdiagnosed.
Symptoms of hATTR amyloidosis vary from person to person, depending on which organs or tissues are affected. As the disease progresses, symptoms may worsen (eg, numbness or tingling of the feet can result in the need to walk with an aid or use a wheelchair) and can lead to significant disability, decreased quality of life, and, in many instances, a shortened lifespan.
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Download the Alnyam ActTM Brochures for hATTR Amyloidosis