I think what you need to understand first is what a disease actually is; a collected set of symptoms with a common cause. In this case, one or more of the symptoms which make up the disease will be caused by the presence of a certain protein in the body. In order to stop these symptoms (remember, the symptoms are the disease) the best way is to reduce the quantity of this protein. In order to reduce the quantity of the protein, siRNA is used to prevent it's translation and so prevent further production. Less Protein = Less symptoms
Hope this helps.