Ten are for rare or “orphan” diseases, which frequently lack any therapy because of the small number of patients with the condition, such as a treatment for hereditary angioedema;
十种药是为罕见或“孤儿”病，因为患者少的情况经常缺乏任何治疗，例如遗传性血管水肿治疗。
2
Gene therapy can be used for hereditarydiseases, but also for other diseases that affect heart, brain and even for cancer.