Spain develops an effective gene therapy against a serious inherited childhood disease
The therapy is targeted at subtype A of Fanconi anemia, caused by mutations in the Fanca gene, and its lengthy development ...
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Rare disease day 2025 : What is Fanconi Anemia, a rare blood disorder that calls for urgent ...
Fanconi anemia (FA) is a rare genetic disorder impairing bone marrow's blood cell production, leading to severe health issues. Early diagnosis and treatment, especially through blood stem cell ...