Overview
Hereditary transthyretin-mediated amyloidosis is treated with 1 medication in our database, including TRYNGOLZA. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Alnylam Pharmaceuticals. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hereditary transthyretin-mediated amyloidosis treatment below.
Autosomal dominant
Passed on from just one parent; each child has about a 50% chance of inheriting it
Adult
Begins in adulthood (age 18 or older)
FDA & Trial Timeline
6 eventsAlnylam Pharmaceuticals — PHASE3
AMVUTTRA: FDA approved
treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits
Attruby: FDA approved
treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization
WAINUA: FDA approved
Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Onpattro: FDA approved
ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
6 availableVyndaqel
indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospit…
indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization
AMVUTTRA
treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart f…
treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits
Attruby
treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization
WAINUA
Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Onpattro
ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
TRYNGOLZA
* Patient Copay Amount: As little as $0 per fill for commercially insured patients * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Available to commercially ins
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
5 resourcesThe Assistance Fund — Hereditary transthyretin-mediated amyloidosis
The Assistance Fund
Hereditary transthyretin-mediated amyloidosis
Travel Grants
No travel grants are currently matched to Hereditary transthyretin-mediated amyloidosis.
Community
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Start the conversation →Latest news about Hereditary transthyretin-mediated amyloidosis
3 articlesCaregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Hereditary transthyretin-mediated amyloidosis
What is Hereditary transthyretin-mediated amyloidosis?
Hereditary transthyretin-mediated amyloidosis is treated with 1 medication in our database, including TRYNGOLZA. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Alnylam Pharmaceuticals. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hereditary transthyretin-mediated amyloidosis treatment below.
How is Hereditary transthyretin-mediated amyloidosis inherited?
Hereditary transthyretin-mediated amyloidosis follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Hereditary transthyretin-mediated amyloidosis typically begin?
Typical onset of Hereditary transthyretin-mediated amyloidosis is adult. Age of onset can vary across affected individuals.
Are there clinical trials for Hereditary transthyretin-mediated amyloidosis?
Yes — 1 recruiting clinical trial is currently listed for Hereditary transthyretin-mediated amyloidosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Hereditary transthyretin-mediated amyloidosis?
15 specialists and care centers treating Hereditary transthyretin-mediated amyloidosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Hereditary transthyretin-mediated amyloidosis?
1 FDA-approved treatment and 4 patient support programs are currently tracked on UniteRare for Hereditary transthyretin-mediated amyloidosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.