使用Player FM应用程序离线!
Advancing a Gene Therapy for a Rare and Fatal CNS Disorder
Manage episode 359431613 series 60790
GM1 gangliosidosis is a rare and deadly lysosomal storage disorder that causes progressive damage to neurons in the brain, as well as the heart, liver, bones and other tissues throughout the body. There are currently no approved therapies to treat the condition. Passage Bio, which has a collaboration with the Gene Therapy Program at the University of Pennsylvania, is developing a gene therapy to the condition. We spoke to Samiah Al-Zaidy, vice president of clinical development for Passage, about GM1 gangliosidosis, the company’s experimental therapy to treat the condition, and what’s known about the therapy from work that’s been done to date.
487集单集
Manage episode 359431613 series 60790
GM1 gangliosidosis is a rare and deadly lysosomal storage disorder that causes progressive damage to neurons in the brain, as well as the heart, liver, bones and other tissues throughout the body. There are currently no approved therapies to treat the condition. Passage Bio, which has a collaboration with the Gene Therapy Program at the University of Pennsylvania, is developing a gene therapy to the condition. We spoke to Samiah Al-Zaidy, vice president of clinical development for Passage, about GM1 gangliosidosis, the company’s experimental therapy to treat the condition, and what’s known about the therapy from work that’s been done to date.
487集单集
Alle afleveringen
×欢迎使用Player FM
Player FM正在网上搜索高质量的播客,以便您现在享受。它是最好的播客应用程序,适用于安卓、iPhone和网络。注册以跨设备同步订阅。