Biotech Values

[ghmm]

BMRN / Rare diseases

There is a Biomarin Movie (30 minutes) about the disease/development/regulatory process for Brineura which was approved to treat the CLN2 form of Batten disease. I thought was excellent from just about every aspect. I suspect Biomarin was involved in the making and some may see it as biased, though I think its still worthwhile.

It starts off talking about the disease and the families. Its such a terrible disease that should give some perspective to investors on what the impact is on those with these types of diseases and their families.

It talks a little about how the drug was developed and how the program kept going, then about the filing and regulatory process with some insights I wasn't aware of and how important it was to have patient advocacy for the approval of the drug.

The video is freely available on YouTube