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Does Anyone With Familial ATTR Have Both Liver And Cardiac Involvement? What Has Been Your Treatment Protocol, Progression ? Thank You!

A MyAmyloidosisTeam Member asked a question 💭
Philadelphia Area, PA
June 15
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A MyAmyloidosisTeam Member

Yes, individuals with familial ATTR (hereditary ATTR amyloidosis) can have both liver and cardiac involvement. The liver produces abnormal TTR proteins, which can lead to amyloid deposits in various organs, including the heart. This can result in cardiac amyloidosis, which affects the heart's ability to pump blood properly.

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Yes, individuals with familial ATTR (hereditary ATTR amyloidosis) can have both liver and cardiac involvement. The liver produces abnormal TTR proteins, which can lead to amyloid deposits in various organs, including the heart. This can result in cardiac amyloidosis, which affects the heart's ability to pump blood properly.

Treatment Protocols:
1. Medications:
- Tegsedi (inotersen) and Onpattro (patisiran): Approved by the FDA for hereditary ATTR in 2018.
- Tafamidis (Vyndamax, Vyndaqel): Approved in 2019, shown to slow disease progression and reduce cardiac-related hospitalization and death.

2. Liver Transplantation:
- Effective for hereditary ATTR amyloidosis as it removes the source of abnormal TTR proteins, potentially slowing or stopping disease progression.

3. Heart Transplantation:
- Sometimes combined with liver transplantation for severe cases.

4. Gene-Silencing Therapies:
- These interfere with genetic instructions to reduce the production of abnormal TTR proteins.

Progression:
- The progression of familial ATTR amyloidosis can vary based on the specific mutation and the organs involved. Treatments like tafamidis have been shown to slow progression and improve quality of life.

Always consult with your healthcare provider to determine the most appropriate treatment plan for your specific condition.

June 15

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