Described herein are pharmaceutical compositions and methods for treating and preventing conformational diseases such as, TDP-43 proteinopathies, SMA, amyloid positive cancer, normal and premature aging. Also disclosed arein vitroscreening methods for screening a therapeutic candidate to treat conformation diseases, by measuring the expression level of prion-like folding of aggregation-prone proteins or a P53 aggregate.本文所描述的是用於治療及預防如TDP-43蛋白質病變、SMA、類澱粉陽性癌症、正常及過早老化(premature aging)的構形疾病之醫藥組合物及方法。同時亦揭露藉由測量聚集傾向蛋白的普里昂樣摺疊或P53聚集體的表現量以篩選用於治療構形疾病之治療候選物的體外篩選方法。
