Overview
Beta Thalassemia is treated with 3 medications in our database, including AQVESME, AURLUMYN, Reblozyl. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Agios, Eicos, Bristol Myers Squibb. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Beta Thalassemia 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
Infantile
Begins in infancy, roughly 1 month to 2 years old
FDA & Trial Timeline
10 eventsHospital Clinic of Barcelona — NA
Assiut University
Bristol-Myers Squibb
AQVESME: FDA approved
treatment of anemia in adults with alpha- or beta-thalassemia
University Research Institute for the Study of Genetic & Malignant Disorders in Childhood
Assiut University
Centre Hospitalier Metropole Savoie — NA
Tanta University — PHASE3
Hu Peng — NA
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
3 availableReblozyl
REBLOZYL is indicated for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions
AQVESME
treatment of anemia in adults with alpha- or beta-thalassemia
AURLUMYN
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
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
2 resourcesTravel Grants
No travel grants are currently matched to Beta-thalassemia.
Community
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Start the conversation →Latest news about Beta-thalassemia
Disease timeline:
New recruiting trial: Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia
A new clinical trial is recruiting patients for Beta-thalassemia
New recruiting trial: Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
A new clinical trial is recruiting patients for Beta-thalassemia
New recruiting trial: Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
A new clinical trial is recruiting patients for Beta-thalassemia
New recruiting trial: Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia
A new clinical trial is recruiting patients for Beta-thalassemia
New recruiting trial: An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease
A new clinical trial is recruiting patients for Beta-thalassemia
New recruiting trial: ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
A new clinical trial is recruiting patients for Beta-thalassemia
New recruiting trial: Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
A new clinical trial is recruiting patients for Beta-thalassemia
New recruiting trial: Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
A new clinical trial is recruiting patients for Beta-thalassemia
New recruiting trial: Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
A new clinical trial is recruiting patients for Beta-thalassemia
New recruiting trial: Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
A new clinical trial is recruiting patients for Beta-thalassemia
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 Beta-thalassemia
What is Beta-thalassemia?
Beta Thalassemia is treated with 3 medications in our database, including AQVESME, AURLUMYN, Reblozyl. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Agios, Eicos, Bristol Myers Squibb. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Beta Thalassemia treatment below.
How is Beta-thalassemia inherited?
Beta-thalassemia follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Beta-thalassemia typically begin?
Typical onset of Beta-thalassemia is infantile. Age of onset can vary across affected individuals.
Are there clinical trials for Beta-thalassemia?
Yes — 20 recruiting clinical trials are currently listed for Beta-thalassemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Beta-thalassemia?
25 specialists and care centers treating Beta-thalassemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Beta-thalassemia?
3 FDA-approved treatments and 2 patient support programs are currently tracked on UniteRare for Beta-thalassemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.