Extraordinary Measures

The movie "Extraordinary Measures" opened this weekend.  Matt and I plan on seeing it soon.  It is a true story of a family with 2 children who have Pompe Disease.  It is a disease similar to Sanfilippo.  They are both Lysosomal Storage Disorders.  This movie highlights the difficulties families face in getting a treatment option.  Because these are orphan diseases (i.e. rare), they do not get funding.  Without funding researchers and doctors are unable to carry out their research.  And pharmaceutical companies don't give them the attention they need because they won't receive a huge payoff for their work, since there aren't a lot of children who will need the treatment.

I hope this movie celebrated the accomplishments of those involved in finding a treatment for Pompe.  I also hope it serves as a reminder that there are many other similar diseases without treatments and children are dying.

http://extraordinarymeasuresthemovie.com/