This causes serious, wide-rangingsymptoms, including heart disease, kidney failure, and nerve damage.
Promising treatments have been approved and more are undergoing clinical trials, including ATTR-CM treatments.
TTR Stabilizers
TTR stabilizers are drugs that stop the TTR protein from creating amyloid deposits.
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These deposits are what cause many of the symptoms of hATTR.
TTR Silencers
The second class of medications are gene silencers.
They stop TTR production by silencing the gene that causes the protein to form and build up.
These are called supportive therapies, because they support you in living a healthier, more comfortable lifestyle.
However, a liver transplant is still an option for patients who dont respond to treatments.
Because the TTR protein is produced in the liver, a transplant may help stop production.
Yet, some patients continue to have irregular TTR production even with a new liver.
Before you do that, talk with your healthcare provider.
Your healthcare provider can also suggest lifestyle changes, such as provider-approved exercise, that might help manage symptoms.
Working with a physical therapist or dietitian familiar with hATTR and your symptoms might also help.
Consider seeing a counselor, or joining a support group for people with amyloidosis.Self-carecan play an important role too.
Participating in clinical trialscan open up new treatment options for patients with hATTR.
Talk with your healthcare provider about the benefits and risks of participating in clinical trials.
Summary
Hereditary transthyretinamyloidosiscan be fatal, but there have been marked improvements in treatment in recent years.
Ther Clin Risk Manag.
- doi: 10.2147/TCRM.S219979
Amyloidosis Research Consortium.Hereditary transthyretin amyloidosis.
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Amyloidosis Research Consortium.Inotersen (Tegsedi) factsheet.
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