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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.
News
The first human model for neurodegenerative Sanfilippo syndrome
Researchers from the Institut Pasteur and Inserm, in collaboration with the Centre de Biologie Cellulaire in Lyon, have obtained the first human neuron model for Sanfilippo syndrome, an incurable neurodegenerative disorder that occurs in children. This model is the tool of choice for studying the cellular mechanisms at the root of this disease as well as for identifying therapeutic options. The procedure, which involves stem cells, could also be applied to model other diseases, especially neurodegenerative diseases. This work has been published in the Human Molecular Genetics journal.
RARE 2011 - Rare and Orphan Diseases Colloquium (Montpellier, 2-4 nov. 2011)
‘‘Innovation and partnerships for patients’’
This conference initiated and organized by the Eurobiomed Competitivness Cluster aims to promote partnerships between the key players in research and industry.
Researchers, industrial companies and patient organizations will meet on this occasion around workshops, conferences and partnership meetings.
An international day dedicated to the European actors in the field of rare diseases.
Programme and registration
US Conference on Rare diseases and Orphan products (11 – 13 Oct. 2011 Washington DC)
Separate tracks for patients, researchers, companies, and investors, will be organized in addition to plenary sessions where leading researchers, company officials, patient organizations and government leaders will discuss how the various interests can collaborate more productively.