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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.
News
The 6th European Conference on Rare Diseases & Orphan Products - 23 to 25 May 2012 - Brussels, Belgium
Each year, this conference provides the opportunity for all actors involved in the fight against rare diseases (patients, families, healthcare professionals, industry, researchers, etc..) to meet and exchange information and ideas. The 2012 edition will focus on the latest scientific advances in treatments and innovations in healthcare, social care and support.
On Wednesday 23 May 23, the General Assembly of EURORDIS will be organized for its members followed by a series of forums and workshops for capacity building. These sessions are designed to empower patients and patients’ advocates and encourage learning from each other’s experience.
Alliance SANFILIPPO will be there: Karen Aiach will participate to the session "New trends in collaborations between industry, academia and patient organizations" on Wednesday 23th May, 5:00 pm.
12th International symposium on MPS and related diseases, Noordwijkerhout, the Netherlands, from 28 June to 1 July 2012.
This symposium aims to allow exchanges between patients, parents, families, doctors and scientists on all aspects of mucopolyssaccharidosis and related disorders. Families will not only have opportunities to meet peers from other countries, but will be able to attend sessions on optimizing care, dealing with clinical issues and surgery in MPS, and ‘living fully with MPS’. Scientific sessions will focus on musculoskeletal disease and MPS, the brain and MPS, and new approaches to treatment.
Programme and registration on http://www.mps2012.eu
French Fondation de cooperation scientifique Maladies Rares was officially launched
On the occasion of the Rare Diseases Day 2012, the French Fondation de cooperation scientifique Maladies Rares was officially launched. During the press conference, Nora Berra in charge of Health welcomed the new structure as "a model of multidisciplinary and interdepartmental cooperation. It is a strike force to advance research."
"Research is still suffering from lack of coordination on sources and use of data, and from fragmentation of the research teams and research programs. These weaknesses are mainly due to the dispersion of structures dedicated to rare diseases, "said Prof. Nicolas Levy, director of the Foundation.
Partners are multiple : French Association Against the Myopathies - a major-league player in France's rare disease field and organisers of the country's famously successful Telethon; national medical and health research organisation INSERM; the Conference of General Directors of the University Hospitals and the Conference of Presidents of the University.
The foundation will act with all public and private actors because the objective is to bring together all the skills in the fight against these diseases.
See the website : http://fondation-maladiesrares.org/
Rare Didease Day 2012 : "Rare but strong together"
In its fifth year, Rare Disease Day will seek to draw attention to the importance of solidarity. Solidarity and collaboration amongs patients, families, all stakeholders and at global level. Many events will take place in over 50 countries. More information on the dedicated website www.rarediseaseday.org
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.
See the Press release (2011, 13sept.)
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.
Family Higonnet seeks to bring together families affected by the type C of the Sanfilippo disease worldwide.
The MPS IIIC is a particularly rare form of the disease and the family Higonnet member of Alliance Sanfilippo, has created an association partner, SANFILIPPO SUD, which aims to bring together those concerned. Research teams are working in this area (see in particular the MPS IIIC program of Alliance Sanfilippo ) and families want to be involved to help them in their projects.
Famille Higonnet :
Sanfilippo Sud
Lieu dit Les Planes
82210 SAINT ARROUMEX
France
Email : sanfilippo-sud@orange.fr
Tel: +33 (0) 6 07 15 36 60
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)