Hello NNPDF Families and Friends,
The link below will allow you access and view a YouTube video developed by Genzyme Pharmaceuticals (A Sanofi Company), on how the scientists and researchers at Genzyme develop and produce therapeutic medications for rare diseases. The process is simply as fascinating as it is hard to believe. This information is very timely, especially as both our our NPD communities, Niemann-Pick Disease Type C and NPD Type A&B (ASMD), are both involved in clinical trials at the moment. I hope that you find this of interest.
Nadine Hill; NNPDF Executive Director
"By inserting into living mammalian cells a human gene that directs the production of a particular enzyme, we essentially turn those cells into tiny "factories" that produce the enzyme, which can then be harvested and purified for medical use in humans."