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The gene therapy is specifically approved for those patients who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or repeated, serious spontaneous bleeding episodes and do not have neutralizing antibodies to the adeno-associated virus serotype Rh74var (AAVRh74var) capsid

2,13 Liver biopsy is the only test that distinguishes hepatic steatosis from nonalcoholic steatohepatitis, but its routine use is controversial in persons with nonalcoholic fatty liver disease

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The escalation protocol feature allows you to build custom dose ramp-ups and see the pharmacokinetic consequences before committing to a schedule