Gaurav Shah, MD, Chief Executive Officer and President of Rocket Pharmaceuticals, explains Fanconi anemia, a rare blood disorder that his company is developing a gene therapy to treat.

Fanconi anemia is an inherited disorder involving mutations of one of many possible FANC genes.

Persons with Fanconi anemia have prone to bone marrow failure, acute myeloid leukemia, solid tumors, and developmental abnormalities. Classic features may include abnormal thumbs, absent radii, short stature, skin hyperpigmentation, microcephaly, abnormal kidneys, and decreased fertility. Current treatment options are limited to allogeneic hematopoietic stem cell transplantation.

To learn more about this and other rare blood disorders, visit



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