Overview
Hereditary Amyloidosis is treated with 3 medications in our database, including AMVUTTRA, Onpattro, WAINUA. 3 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Alnylam, AstraZeneca. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hereditary Amyloidosis treatment below.
Variable
Can be inherited in different ways depending on the underlying gene
Adult
Begins in adulthood (age 18 or older)
FDA & Trial Timeline
10 eventsUniversity Hospital, Montpellier — NA
Mayo Clinic
Virginia Commonwealth University
University of Texas Southwestern Medical Center
Seoul St. Mary's Hospital — PHASE4
Eidos Therapeutics, a BridgeBio company — PHASE3
Qilu Pharmaceutical Co., Ltd. — PHASE4
Yale University
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
3 availableOnpattro
* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Patients must have commercial insurance and be residents of the U
AMVUTTRA
* Patient Copay Amount: $0 for most eligible patients * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Must have commercial insurance; not eligible for patients
WAINUA
* Patient Copay Amount: As little as $0 per month * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Must have commercial insurance, a valid prescription for WAINU
Rare Disease Specialist
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
Travel Grants
9 grantsAstraZeneca Access 360
AstraZeneca
Access 360 provides support services for US patients prescribed FASLODEX to help navigate insurance coverage and out-of-pocket cost concerns. The program offers resources for patient affordability and assistance with accessing AstraZeneca medications.
AstraZeneca Oncology Patient Transportation Program
AstraZeneca
Good Days provides financial and planning assistance for ground and air transportation, lodging, and parking for patients traveling at least 50 miles for medical care. Eligibility is based on medical necessity as determined by the patient's prescribing physician.
Community
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Start the conversation →Latest news about Hereditary amyloidosis
Disease timeline:
New recruiting trial: To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy
A new clinical trial is recruiting patients for Hereditary amyloidosis
New recruiting trial: Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart Transplantation
A new clinical trial is recruiting patients for Hereditary amyloidosis
New recruiting trial: Exploring Biomarkers in Hereditary Transthyretin Amyloidosis
A new clinical trial is recruiting patients for Hereditary amyloidosis
New recruiting trial: Comprehensive Program for Hereditary Transthyretin Amyloidosis
A new clinical trial is recruiting patients for Hereditary amyloidosis
New recruiting trial: PATHWAY-RCT: Preventing Admission To Hospital With Attr cardiomyopathY
A new clinical trial is recruiting patients for Hereditary amyloidosis
New recruiting trial: NatiOnal Referral cenTEr Study of Transthyretin Amyloid Cardiomyopathy(ATTR) Patients on Tafamidis
A new clinical trial is recruiting patients for Hereditary amyloidosis
New recruiting trial: Phenotyping and Characterization of wtATTR-CM (TRACE 1)
A new clinical trial is recruiting patients for Hereditary amyloidosis
New recruiting trial: Dominantly Inherited Alzheimer Network (DIAN)
A new clinical trial is recruiting patients for Hereditary amyloidosis
New recruiting trial: Registre HEAR, Healthcare European Amyloidosis Registry
A new clinical trial is recruiting patients for Hereditary amyloidosis
New recruiting trial: Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers
A new clinical trial is recruiting patients for Hereditary amyloidosis
Caregiver 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 amyloidosis
What is Hereditary amyloidosis?
Hereditary Amyloidosis is treated with 3 medications in our database, including AMVUTTRA, Onpattro, WAINUA. 3 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Alnylam, AstraZeneca. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hereditary Amyloidosis treatment below.
At what age does Hereditary amyloidosis typically begin?
Typical onset of Hereditary amyloidosis is adult. Age of onset can vary across affected individuals.
Are there clinical trials for Hereditary amyloidosis?
Yes — 4 recruiting clinical trials are currently listed for Hereditary 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 amyloidosis?
9 specialists and care centers treating Hereditary 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 amyloidosis?
3 FDA-approved treatments and 1 patient support program are currently tracked on UniteRare for Hereditary amyloidosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.