Cellenkos gains FDA orphan drug status for CK0804 to treat myelofibrosis
The condition has an estimated prevalence of 25,000 patients in the US and an annual incidence of one to three new cases per 100,000 people. CK0804 is an
Denali’s lead investigational TransportVehicle-enabled enzyme replacement therapy is intended to treat mucopolysaccharidosis type II (Hunter syndrome, a rare genetic disorder affecting boys). The US Food and Drug Administration