AVXS
AveXis Inc
stock NASDAQ

Sounds like you know nothing about the studies involving rare, genetic diseases, and how sometimes because of the low patient population you need surrogate endpoints. I remember comments like this when AVXS was studying a gene therapy for spinal muscular atrophy without a control group.(using known course of the disease instead for ethical reasons.) Now that drug is FDA approved, and saving lives. By the way both drugs are from the same researcher, Dr. Jerry Mendel, and use the same AAV capsid delivery mechanism. Only thing different is the gene being delivered. Oh, and the company is researching heavily and partnering with other companies on developing delivery mechanisms that don’t require AAV capsids. This is the first gene therapy for Duchenne muscular dystrophy, so its first generation and they will develop something better. But you know what, right now nothing else exists to help these kids. Oh by the way gene therapy only needs to be done once because the body begins replicating the gene.

Sounds like you know nothing about the studies involving rare, genetic diseases, and how sometimes because of the low patient population you need surrogate endpoints. I remember comments like this when AVXS was studying a gene therapy for spinal muscular atrophy without a control group.(using known course of the disease instead for ethical reasons.) Now that drug is FDA approved, and saving lives. By the way both drugs are from the same researcher, Dr. Jerry Mendel, and use the same AAV capsid delivery mechanism. Only thing different is the gene being delivered. Oh, and the company is researching heavily and partnering with other companies on developing delivery mechanisms that don’t require AAV capsids. This is the first gene therapy for Duchenne muscular dystrophy, so its first generation and they will develop something better. But you know what, right now nothing else exists to help these kids. Oh by the way gene therapy only needs to be done once because the body begins replicating the gene.