Fanconi Anemia
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News
REGULATORY NEWS | EMA Accepts Rocket Pharma’s Gene Therapy Application for Fanconi Anemia
RP-L102 is a gene therapy containing autologous hematopoietic stem cells that have been modified to contain a functional copy of the FANCA gene. Mutations in FANCA hinder DNA repair and occur…
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News
CLINICAL NEWS | Promising Outcomes Found with Stem Cell Transplant for Fanconi Anemia-related Leukemia, MDS
Two-thirds of patients who received allogeneic hematopoietic stem cell transplantation (allo-HSCT) to treat myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) resulting from Fanconi anemia (FA) were still alive at…
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News
NEW TREATMENTS | Gene Editing, mRNA Hold Promise as Fanconi Anemia Therapies
That is the major finding of a newly published review that examined how a better understanding in the scientific and molecular underpinnings of the disease has translated into potential new…
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News
SCIENCE NEWS | Research On Rare Genetic Disease Sheds Light On A Common Head And Neck Cancer
Patients with the rare genetic disorder Fanconi anemia lack certain elements of this repair system, making their cells incapable of removing lesions created by DNA-damaging chemicals, called aldehydes. And…
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