Resources for More Information
Below are links to more information about beta-thalassemia, gene therapy and clinical research studies for patients, caregivers and healthcare professionals.
U.S. Government Resources
Learn more information and view resources on clinical research studies.
Read up on gene therapy in a comprehensive handbook on genetic research.
Patient Group Resources
The American Society of Gene and Cell Therapy’s mission is to advance research and education leading to the discovery and application of gene and cell therapies. The society brings together stakeholders to incorporate the use of gene and cell therapies to alleviate human disease.
Be the Match educates patients and families on marrow transplants for immune system and genetic disorders.
Cooley’s Anemia Foundation’s mission is to advance the treatment and cure for various forms of thalassemia, including beta-thalassemia major. The foundation works to enhance patients’ quality of life and to educate medical professionals and those that may carry the genetic trait for beta thalassemia.
Global Genes is one of the leading rare disease patient advocacy organizations in the world. Global Genes has many resources that equip advocates to become activists for their disease, including a toolkit that provides rare disease patients and caregivers with an overview of gene therapy and why it is being considered as a possible treatment for many rare diseases.
The National Organization for Rare Disorders (NORD) is a non-profit federation of voluntary health organizations dedicated to helping people with rare or orphan diseases. NORD is committed to identifying, treating and curing rare disorders through education, advocacy, research and service.
The Thalassemia International Federation’s (TIF) mission is to develop and establish programs to aid in the prevention and quality treatment of thalassemia and other hemoglobin disorders. In particular, TIF works to provide resources to countries where frequency, incidence and prevalence of these disorders are high.
The Center for Information and Study on Clinical Research Participation (CISCRP) is a non-profit organization to educate and inform the public, patients, healthcare professionals, media and policymakers on clinical research. Visit the CISCRP Education Center
for comprehensive resources about clinical research and what it means to participate. Resources posted here include stories
and questions to ask
Additional Resources from bluebird bio
Introduces what genes are and the role they play in genetic diseases.
Provides an overview of the gene therapy approach bluebird bio is researching.
Explains the gene therapy approach for beta thalassemia that bluebird bio is researching.