For instance, from the manufacturer's point of view, an «improvement in the safety of health care» and a «better integration of patients with
hemophilia into a normal social life» already arise through market entry.
Not exact matches
A Shire buy would give it a way
into the
hemophilia market, as well as more drugs for combatting cancer and other illnesses.
Moreover, the results may transcend the bloating and discomfort of lactose intolerance: The technique could serve as a way to transform the gut lining
into a factory for replacing missing proteins in other genetic disorders such as
hemophilia, says pulmonologist Eric Alton of the Imperial College School of Medicine in London.
These multigene interactions are subtler and knottier than the single gene drivers of diseases such as
hemophilia and cystic fibrosis; spotting them calls for statistical inspiration and rigorous experiments repeated again and again to guard against introducing unproven gene tests
into the clinic.
Her
hemophilia patients, like most of us, carried antibodies to AAV, and their immune systems flew
into high gear when they received the engineered virus.
To verify that the process worked, the endothelial cells with the inversion - corrected genes were transplanted
into F8 deficient mice (mice with
hemophilia A) and the mice started producing the F8 clotting factor on their own, which essentially cured them of
hemophilia A.
Concerns about immunological consequences of rAAV - mediated gene transfer were substantiated by the outcome of a clinical trial in which human
hemophilia B patients were infused
into the liver with rAAV -2-F.
We entered
into an agreement with Bayer to research, develop and commercialize AAV gene therapy products for treatment of
hemophilia A.