“THERE IS A CLEAR PATHWAY.”
https://bit.ly/HelpSebNow
Update post Allievex liquidation process.
This is an ethical call to correct a wrong: https://www.facebook.com/Helpsebnow/
My son Sebastien is 11 years old. He has a rare neurodegenerative and very life limiting condition, Sanfilippo. This syndrome causes children to start losing their cognitive and physical skills as early as three years old and typically die in their teens. They lack an essential enzyme (that breaks down Heparan Sulfate). 
Sebastien is reaching the end of the plateau period typical to this condition, a point when much more rapid degeneration takes its course with further loss of his faculties, and a point of no return (seizures, loss of vision, hearing, losing the ability to swallow etc)...equal to slow neurological regression and a short but miserable life. 
This is about Sebastien being at a crucial age of development where we can still save a lot of his capabilities.
A recent treatment tralesinidase alfa (AX 250), an enzyme replacement therapy (ERT) that helps reduce the toxic metabolic compound Heparan sulphate, which in turn builds up in the brain and body of Sanfilippo children, was in the course of being trialed and tested in a major international study and appeared to be successful.
After a 7 year battle to try and get Sebastian onto a recent study of ERT in vain, via an accelerated access scheme, the trial has now been terminated. Yet again, the traditional approval process proved to unnecessarily delay the gateway to effective treatments for patients with “serious progressive diseases” in a world that is already essentially about enterprise, profit and less about lives. 
It is wrong to continue to refuse these children access to products for which there is sufficient safety and efficacy data, together with a chance for a better quality of life, however long that may be. 
However, thanks to tireless advocating from parents, clinicians, lawyers, charities, community, Eurordis, EMA, MHRA as well as the FDA and the various studies involved, there may be some progress towards potential encouragement by the FDA to apply for accelerated approval. We must keep the momentum going. 
Please sign, keep sharing our petition and follow us, in order to maintain visibility, awareness and above all to help continue to convince the FDA and the pharmaceutical world to work hand in hand URGENTLY and grant access to drugs NOW that have already generated clinical evidence of safety and efficacy in order to prevent this generation and the following generation of San Filippo children from being abandoned. 
This is about Sebastien being at a crucial age of development where we can still save a lot of his capabilities.  
There are treatments that are beneficial to Sanfilippo NOW. 
“THERE IS A CLEAR PATHWAY.”
https://bit.ly/HelpSebNow