RP-A501 is an AAV-based gene therapy aimed to restore the LAMP-2 gene which is defective in male patients afflicted with<unk>  and how to cure it. Cardiac transplantation has been performed as a treatment; however, most patients die early in life.
Danon disease
Kufs disease
Ullrich congenital muscular dystrophy
Emery–Dreifuss muscular dystrophy
Degos disease
Pickardt syndrome
Lafora disease
Fabry disease