Hereditary ATTR (hATTR) Amyloidosis: A Rare, Inherited, Rapidly Progressive Condition That Impacts the Nerves, Heart, and Digestive System
June 28, 2023
Hereditary ATTR (hATTR) amyloidosis is a rare, inherited, rapidly progressive disease caused by variants, or changes, in the transthyretin, or TTR, gene. These variants cause the TTR protein to take on an abnormal shape and build up as amyloid deposits in various parts of the body, including the nerves, heart, and digestive system, which cause symptoms of the disease.
Symptoms of hATTR amyloidosis vary widely among people with the condition, even within families, and different symptoms may appear at different times for each person. Common symptoms include tingling and/or numbness in the hands, feet, arms and legs, carpal tunnel syndrome, diarrhea, nausea or vomiting, fatigue, and shortness of breath. Nerve damage that affects sensation, movement, strength, the digestive system, and other bodily functions may be referred to as polyneuropathy, which can lead to significant disability and dysfunction. This is not a complete list of symptoms that may be experienced with hATTR amyloidosis.
Misdiagnosis or delays in diagnosis are common, as symptoms can be similar to those of more common conditions, and awareness is low due to its rarity. However, early diagnosis and management are important as the disease can progress rapidly and significantly. Also, as a genetic disease, when a diagnosis in one patient is delayed, an entire family can remain unaware that they may also be at risk for developing symptoms of the disease.
We’ll sit down with Dr. Robert Gottlieb, an expert in hATTR amyloidosis, to learn about AMVUTTRA® (vutrisiran), a treatment option approved by the U.S. FDA in 2022 for the polyneuropathy of hATTR amyloidosis in adults, as well as John, who was diagnosed with the polyneuropathy of hATTR amyloidosis after his father passed away from the condition, and today is being treated with AMVUTTRA. Please continue reading for the Important Safety Information and Indication for AMVUTTRA.
If you or a family member are experiencing symptoms associated with hATTR amyloidosis, or if you have a family history of the condition, you should talk to your doctor.
Learn more: www.amvuttra.com
Important Safety Information
What are the most important things I should know about AMVUTTRA?
AMVUTTRA can cause:
- Low Vitamin A Levels
Treatment with AMVUTTRA lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day. You should not take more than the amount of vitamin A recommended by your doctor.
Low vitamin A levels can affect vision. If you have problems with your vision (e.g., night blindness) while taking AMVUTTRA, talk to your doctor. Your doctor may refer you to an eye specialist.
What are the common side effects of AMVUTTRA?
The most common side effects of AMVUTTRA are pain in the arms or legs, pain in the joints (arthralgia), shortness of breath (dyspnea), and low vitamin A levels.
These are not all the possible side effects of AMVUTTRA. Talk to your doctor about side effects that you experience. You are encouraged to report negative side effects of prescription drugs to the U.S. Food and Drug Administration (FDA). Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
What is AMVUTTRA® (vutrisiran)?
AMVUTTRA is a prescription medicine that treats the polyneuropathy caused by an illness called hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). AMVUTTRA is used in adults only.
Please see the full Prescribing Information.
Sponsored by Alnylam Pharmaceuticals, Inc. For U.S. residents only.
AMVUTTRA and its associated logo are trademarks of Alnylam Pharmaceuticals.
© 2023 Alnylam Pharmaceuticals, Inc. All rights reserved. AMV-USA-00578
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