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Glossary Library

01/27/11

Alliance SANFILIPPO strategic programs

© Inserm, P. Latron

Alliance SANFILIPPO has been actively involved in research since it was founded.  Its knowledge of the disease and of the experience of children and their families, along with a good understanding of the stakes involved in research, have made it an important partner for the scientific community. 

 

In addition, the Alliance has demonstrated its commitment and its dynamism.  Over 3 million euros (US$3.5 million) were allocated to research between 2006 and 2010, making Alliance SANFILIPPO a key player in MPS III research funding by families.

 

ASF supports 4 main strategic programs:


Read-through of premature stop codon


Anti-inflammatory treatments to decrease the neuroinflammatory process


Gene therapy

 

Program for MPS type IIIC (since late 2010).