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Alliance Sanfilippo and Sanfilippo Syndrome. Science must lead to cures! when progress is made in research on Sanfilippo Syndrome, the march toward brain medicine accelerates.
K. et G.AIACH.
<< MPS IIIC program of Alliance SANFILIPPO
The clinical trial of gene therapy SAF-301 for MPS IIIA is launched.
The sponsor LYSOGENE has released the public information note which describes concretely how the trial will take place for children who will participate and for their parents.
See the note (in French)