The genetic basis of Fanconi anemia (FA) is mysterious and complex, as none of the five genes previously associated with the disorder have homology to genes encoding proteins of known function.

In the area of growth control, our recent work is focused on the mTOR pathway that controls multiple aspects of cell growth and homeostasis. Our work on genomic integrity ranges from the Fanconi ...

Analyst Whitney Ijem from Canaccord Genuity maintained a Buy rating on Rocket Pharmaceuticals (RCKT – Research Report) and increased the ...

Rocket Pharmaceuticals is also progressing in its other projects, like the rolling BLA for Fanconi anemia and the Phase 1 enrollment completion in PKP2-arrhythmogenic cardiomyopathy. Furthermore, the ...

Fanconi anemia has no known cure and can cause bone marrow failure and cancer. So Jimbo and his ex-wife, Candi Fisher, established the Kidz 1st Fund in 2011 to help spread awareness and raise ...

People of Ashkenazi Jewish descent are at higher risk of developing pancreatic cancer because they are more likely than other ...

which supports children affected by fanconi anemia, a rare genetic disorder affecting bone marrow. "It was really fun, we had a few direction issues but we made it there in the end," Dexter said.

RP-L102, which the firm designed to treat a form of disease caused by FANCA mutations, is already under review with the European Medicines Agency.

Its clinical program is an LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer.