About ATTR Amyloidosis (Transthyretin Amyloidosis)
ATTR amyloidosis develops when transthyretin protein misfolds and accumulates as amyloid deposits in organs throughout the body. In hereditary ATTR, a genetic mutation causes TTR protein instability. In wild-type ATTR, normal protein becomes unstable with age. Cardiac involvement causes restrictive cardiomyopathy, while neurological involvement produces peripheral and autonomic neuropathy.
Diagnosis combines cardiac imaging (echocardiography, cardiac MRI, nuclear imaging), tissue biopsy, genetic testing for TTR mutations, and specialized blood tests. Early detection through screening of at-risk populations is crucial since treatments work best when started early.
Multiple FDA-approved therapies now address ATTR amyloidosis. TTR stabilizers like tafamidis and acoramidis prevent protein misfolding and aggregation. Gene-silencing therapies including vutrisiran and eplontersen reduce TTR production. Ongoing research focuses on TTR depleters that remove existing amyloid deposits.
Common Symptoms of ATTR Amyloidosis (Transthyretin Amyloidosis)
Recognizing the signs of ATTR Amyloidosis (Transthyretin Amyloidosis) early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.
- Shortness of breath and progressive heart failure
- Irregular heartbeat and atrial fibrillation
- Leg swelling (edema) and fluid retention
- Peripheral neuropathy with tingling, numbness, or weakness in feet and legs
- Lightheadedness or fainting from cardiac conduction problems
- Autonomic dysfunction including low blood pressure on standing and GI issues
Who ATTR Amyloidosis (Transthyretin Amyloidosis) Affects
Hereditary ATTR typically emerges in middle age, though onset varies significantly by geographic region and specific mutation. African Americans with the Val122Ile mutation have a 3 to 4% carrier prevalence. Wild-type ATTR predominantly affects males over 60. Both forms progress more rapidly in males and older patients.
Find Your Next Step
Answer a few questions and we'll point you to the right tools and information for where you are right now.
FDA-Approved Treatments for ATTR Amyloidosis (Transthyretin Amyloidosis)
There are currently 5 FDA-approved medications for ATTR Amyloidosis (Transthyretin Amyloidosis). These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials. Compare all 5 side by side, with dosing, trial results and warnings from the labels.
Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.
Help Paying for ATTR Amyloidosis (Transthyretin Amyloidosis) Treatment
Charity funds and drugmaker programs for ATTR Amyloidosis (Transthyretin Amyloidosis), checked at the source. Pick your insurance to see what fits.
- From a charity · HealthWell FoundationAmyloidosis fundOpen
Pays for: Copays, premiums or other treatment costs.
- From a charity · HealthWell FoundationCardiomyopathy (Medicare Access) fundOpen
Pays for: Copays, premiums or other treatment costs. Medicare patients only.
- From a charity · NORD RareCareAmyloidosis Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting Applications” - From a charity · NORD RareCareAmyloidosis Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting Applications” - From a charity · The Assistance FundAmyloidosis fundOpen
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “OPEN — Accepting New Patients. TAF is currently accepting new patient enrollments for this program.” - From a charity · TotalAssist (formerly PAN Foundation)Amyloidosis fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $5,500 per year. Requires Medicare, Medicaid or TRICARE.
- Onpattro (Patisiran) · Alnylam Assist
- Amvuttra (Vutrisiran) · Alnylam Assist
- Vyndaqel (Tafamidis) · VynAssist / VyndaLink
- Attruby (Acoramidis) · ForgingBridges
- Wainua (Eplontersen) · WAINUA WAY (AstraZeneca Access 360)
Side Effect Explorer
Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.
Loading side effect data...
Genetic Testing
Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.
The TTR gene page lists every condition Orphanet links to the gene and the open trials that name it.
Finding labs...
Trusted ATTR Amyloidosis (Transthyretin Amyloidosis) Resources
Reputable organizations and medical references for learning more about ATTR Amyloidosis (Transthyretin Amyloidosis), including disease registries, foundation resources, and clinical guidelines.