New orphan drug designation, page-14

Probably a lot more. There’s active National Societies of MPS in Oz and the US.

its a very connected network with annual conferences in Orlando for families and big day to day support networks. PPS and Paradigm’s efforts have already been circulated amongst the community.

I can recall a study done that showed PPS helps normal skeletal (spine & skull), and aorta development if applied early enough in juveniles in animal studies. It was a dramatic difference.

We have a real shot at making a huge difference to that community. Target pharmaceutical companies for partnership would be Biomatin and Sanofi, if any. Such a targeted, well connected community would require little sales effort.