http://www.raredr.com/conferences/worldsymposium/dear-fda-and-pharma
Dear FDA and Pharma - Your Risk Benefit Ratio is Not My Risk Benefit Ratio

Dave Esposito



Dave Esposito, father of a child with Sanfilippo syndrome, wants the FDA and the pharmaceutical industry to know that their definition of success or a good outcome measure is different than a parent's definition.

What is Sanfilippo disease?

Children with Sanfilippo syndrome have inadequate levels of specific enzymes needed to metabolize heparan sulfate. This leads to a build up of heparan sulfate in lysosomes that in turn leads to progressive neural degeneration.
 
There are 4 types of Sanfilippo syndrome based on the specific enzyme that is affected.
 
Sanfilippo Type A: heparan N-sulfatase.
Sanfilippo Type B: alpha-N-acetylglucosaminidase.
Sanfilippo Type C: acetyl-CoAlpha-glucosaminide acetyltransferase.
Sanfilippo Type D: N-acetylglucosamine 6-sulfatase.
 
For more information, visit https://curesff.org/
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