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.
For the heart form, most people are diagnosed without a biopsy: once blood and urine tests rule out AL amyloidosis, a PYP, DPD or HMDP bone scan showing grade 2 or 3 heart uptake confirms ATTR, and TTR gene sequencing separates hereditary from wild-type. The nerve form usually needs nerve testing, a tissue biopsy and genetic testing. A TTR variant alone does not confirm the disease; carriers are monitored, usually starting 10 years before the age their relatives got sick. 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. About 3 to 4% of African Americans carry the Val122Ile (V122I) variant. Wild-type ATTR predominantly affects males over 60.
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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.
ATTR Amyloidosis (Transthyretin Amyloidosis) Medicines in Clinical Trials
Investigational medicines Trial Friend tracks for ATTR Amyloidosis (Transthyretin Amyloidosis), with trial results, safety findings and where each one stands.
Help Paying for ATTR Amyloidosis (Transthyretin Amyloidosis) Treatment
Charity funds and drugmaker programs for ATTR Amyloidosis (Transthyretin Amyloidosis), checked at the source.
- 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 · 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.
- From a charity · The Assistance FundAmyloidosis fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
- Onpattro (Patisiran) · Alnylam Assist
- Amvuttra (Vutrisiran) · Alnylam Assist
- Vyndamax (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.
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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.
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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.