Overview
FDA & Trial Timeline
10 eventsCorrectSequence Therapeutics Co., Ltd — PHASE2
Rongrong Liu — PHASE2
Guangzhou Women and Children's Medical Center — EARLY_PHASE1
Rawan Mohammed Khairy Mostafa Elsawy — NA
Novo Nordisk A/S — PHASE3
Institute of Hematology & Blood Diseases Hospital, China — NA
Rongrong Liu — PHASE2
Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableZynteglo
ZYNTEGLO is indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions.
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
1 resourcesTravel Grants
No travel grants are currently matched to Thalassemia.
Community
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Start the conversation →Latest news about Thalassemia
Disease timeline:
New recruiting trial: A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia
A new clinical trial is recruiting patients for Thalassemia
New recruiting trial: Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
A new clinical trial is recruiting patients for Thalassemia
New recruiting trial: A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
A new clinical trial is recruiting patients for Thalassemia
New recruiting trial: the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia
A new clinical trial is recruiting patients for Thalassemia
New recruiting trial: the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
A new clinical trial is recruiting patients for Thalassemia
New recruiting trial: Evaluation of Recombinant Humanized Anti-CD25 Monoclonal Antibody for Preventing Graft-versus-host Disease After Haploidentical/matched Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Transfusion-dependent Thalassemia
A new clinical trial is recruiting patients for Thalassemia
New recruiting trial: Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients
A new clinical trial is recruiting patients for Thalassemia
New recruiting trial: A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants
A new clinical trial is recruiting patients for 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 Thalassemia
Are there clinical trials for Thalassemia?
Yes — 18 recruiting clinical trials are currently listed for Thalassemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Thalassemia?
25 specialists and care centers treating 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 Thalassemia?
1 patient support program are currently tracked on UniteRare for Thalassemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.