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Functionality was preserved until 2020, when progressive paresthesia and paresis emerged in the left lower limb, later involving the left upper limb, with spasticity (predominantly left-sided) and dysarthria

it protects tissue from further damage by modulating the inflammatory cascade systemically

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

Your provider adjusts the plan based on how your body is responding