Alliance SANFILIPPO’s main objective is to find a treatment which will cure children with mucopolysaccharidosis III and allow them to live in good health.
As of today, there is no cure for this rare disease.  The available treatments can only alleviate the symptoms; they cannot slow the inexorable and dramatic development of the disease.

Scientific research is the only way to reach our goal.  Alliance SANFILIPPO devotes all of its energy, its determination and its professionalism to mobilizing researchers and clinicians, getting them to direct their research towards discovering treatments for our children.

This mobilization is particularly effective because Alliance SANFILIPPO has the unique advantage of using methods from the world of business to support the continuum of biomedical research, facilitating the transfer of new knowledge into clinical research and the production of new treatments.  Thus financial support from Alliance SANFILIPPO concretely turns projects into reality:  in three years Alliance SANFILIPPO has contributed 3 million euros in funding to research programs.  In addition, Alliance SANFILIPPO participates in scientific program management, bringing together experts and energizing progress.  The president of ASF has founded a private law organization, LYSOGENE, which promotes clinical trials and facilitates their realization.

All fields of research are essential and must be developed for MPS III.  Fundamental research gives a better understanding of the molecular mechanisms of the disease, from genetics to physiopathology.  This basic knowledge is crucial to the development of new therapeutic avenues targeting key mechanisms and molecules involved in MPS III.  Translational research enables new scientific ideas to be developed more quickly into therapeutic applications, thereby ensuring a connection between fundamental research and clinical research.  In terms of clinical research, unprecedented developments in biotherapy techniques have allowed researchers to focus on clinical innovations which bring new hope.

A great many disciplines (neurology, biochemistry, epidemiology, human sciences, etc.), along with all of the resources involved in research (biological sample banks, vector production, etc.) are needed to discover effective treatments.

It should also be noted that while patients need researchers, researchers will not find treatments without the collaboration of patients/parents:  their knowledge of the disease and their participation in clinical research are vital. For more (Chap. “Families / Participation in Research”)

Alliance SANFILIPPO acts on behalf of sick children and their families to remind all stakeholders (researchers, industry, health authorities) of the urgent need for effective treatments, and to contribute maximum support to facilitating their discovery and access for all.