teamsanfilippo.blogspot.com
Team Sanfilippo: The Sad Truth About the US Rare Disease Drug Approval Process
http://teamsanfilippo.blogspot.com/2011/02/sad-truth-about-us-rare-disease-drug.html
Tuesday, February 22, 2011. The Sad Truth About the US Rare Disease Drug Approval Process. When we were first found out that our son had elevated urinary GAG levels (a marker for MPS diseases) we assumed he had Hunter Syndrome. Without treatment, Hunter Syndrome is - like all MPS diseases - an absolutely catastrophic disease. When I went to the Shire website to research treatments, I saw the following quote regarding the efficicacy of Elaprase:. Since no treatment is approved, Sanfilippo parents are extr...
teamsanfilippo.blogspot.com
Team Sanfilippo: February 2011
http://teamsanfilippo.blogspot.com/2011_02_01_archive.html
Sunday, February 27, 2011. The Inequity of Rare Disease Knows No Boundary. That is why on International Rare Disease Day, I want to support the efforts of my good friend Simon Ibell. Please take a moment to check out what Simon is doing and help us spread the message:. HEY CANADA, ON FEB. 28TH, BE FAIR TO RARE. Links to this post. Tuesday, February 22, 2011. The Sad Truth About the US Rare Disease Drug Approval Process. In a clinical study of 96 patients with Hunter syndrome, weekly treatment with ELAPRA...