Fanconi anemia
Synonyms: Fanconi pancytopenia
A rare genetic multisystem disorder characterized by progressive pancytopenia with bone marrow failure variable congenital malformations and predisposition to develop hematological or solid tumors.
Data from Orphanet are used to provide information on a disease's name, synonym(s), and overview.
Reference: Access aggregated data from Orphanet at Orphadata.
Orphadata: Free access data from Orphanet. © INSERM 1999. Available on http://www.orphadata.org. Data version April 2024
Newly diagnosed with
Fanconi anemia?
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Advocacy Organizations
DDX3X Foundation
Our mission is to connect families, resources, and the medical community to advance research for a treatment or cure to DDX3X Syndrome. Our ultimate goal is to accelerate brain function in individuals affected by DDX3X Syndrome through advances in cell and gene therapy and pharmaceuticals.
Fanconi Cancer Foundation
The Fanconi Cancer Foundation aims to improve the lives of people affected by FA and associated cancers worldwide by funding exceptional research and empowering our community. We believe that by addressing the core issue of this disease - the inability to repair DNA - we will impact not only people with FA, but anyone affected by cancer.
Clinical Trials
For a list of clinical trials in this disease area, please click here.