Overview
Alpha-1 Antitrypsin Deficiency is treated with 1 medication in our database, including ALBUMINEX. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Bio Products Laboratory. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Alpha-1 Antitrypsin Deficiency treatment below.
Clinical phenotype terms— hover any for plain English:
Autosomal recessive
Passed on when both parents carry the same gene change; often skips generations
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
10 eventsCSL Behring — PHASE4
AIRNA Corporation — PHASE1
University Hospital, Bordeaux
Tessera Therapeutics, Inc. — PHASE1, PHASE2
University of Alabama at Birmingham — PHASE4
Muğla Sıtkı Koçman University
RenJi Hospital — EARLY_PHASE1
Weill Medical College of Cornell University — PHASE1
BioMarin Pharmaceutical — PHASE1
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availableProlastin
For chronic replacement therapy of individuals having congenital deficiency of alpha1- proteinase inhibitor with clinically demonstrable panacinar emphysema.
ALBUMINEX
Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo
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
Financial Resources
5 resourcesHealthWell Foundation — Alpha-1-antitrypsin deficiency
HealthWell Foundation
Alpha-1-antitrypsin deficiency
NORD Patient Assistance — Alpha-1-antitrypsin deficiency
NORD Patient Assistance
Alpha-1-antitrypsin deficiency
Patient Services Inc — Alpha-1-antitrypsin deficiency
Patient Services Inc
Alpha-1-antitrypsin deficiency
PAN Foundation — Alpha-1-antitrypsin deficiency
PAN Foundation
Alpha-1-antitrypsin deficiency
The Assistance Fund — Alpha-1-antitrypsin deficiency
The Assistance Fund
Alpha-1-antitrypsin deficiency
Travel Grants
No travel grants are currently matched to Alpha-1-antitrypsin deficiency.
Community
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Start the conversation →Latest news about Alpha-1-antitrypsin deficiency
5 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 Alpha-1-antitrypsin deficiency
What is Alpha-1-antitrypsin deficiency?
Alpha-1 Antitrypsin Deficiency is treated with 1 medication in our database, including ALBUMINEX. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Bio Products Laboratory. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Alpha-1 Antitrypsin Deficiency treatment below.
How is Alpha-1-antitrypsin deficiency inherited?
Alpha-1-antitrypsin deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Alpha-1-antitrypsin deficiency?
Yes — 20 recruiting clinical trials are currently listed for Alpha-1-antitrypsin deficiency on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Alpha-1-antitrypsin deficiency?
25 specialists and care centers treating Alpha-1-antitrypsin deficiency are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Alpha-1-antitrypsin deficiency?
1 FDA-approved treatment and 6 patient support programs are currently tracked on UniteRare for Alpha-1-antitrypsin deficiency. See the treatments and support programs sections for copay assistance, eligibility, and contact details.