The Rare Diseases Foundation is 10 years old!

A flagship measure of the "Research" axis of the 2nd National Plan for Rare Diseases (2011-2014), a national public health plan led by the Minister of Health, the Rare Diseases Foundation was created on February 7, 2012 by decree of the Minister of Higher Education and Research. This anniversary is an opportunity to measure the magnificent path we have taken and to associate all the actors with whom we work every day: researchers, patient associations, rare disease networks and structures, drug manufacturers, but also all our partners and donors who trust us! You will find below testimonials from our partners on a daily basis.

Our goal: to continue to develop research together to fight rare diseases and bring real solutions to patients and their families!

Mrs. Laurence Tiennot-Herment

President of AFM-Telethon

"For the past 10 years, the Rare Diseases Foundation has structured its actions to support research on rare diseases through calls for projects, but also by supporting associations in their research activities. It also plays an important role in the communication of all this work by multiplying initiatives with the scientific community and the general public. And finally, an original and valuable action, it proposes, via the POC Club, a rapprochement between these academic projects and the industrial world, to enable them to develop and reach the patient.

Mr. Philippe Berta

President of the study group of the National Assembly on rare diseases Member of Parliament for the 6th constituency of the Gard

"The Foundation is a valuable member of the rare disease community. Its commitment and professionalism make it an essential player in the realization of projects, in the funding of research, but also in building bridges between patient associations, the scientific community, biotechs and industry, institutions and the general public. Today we are celebrating the Foundation's 10th anniversary and the crowning of its success, which I hope will continue to benefit our 3 million patients for a long time to come!

Pr Veronique Paquis-Flucklinger

Head of the "Mitochondria, diseases and aging" team at IRCAN, Inserm U1081, CNRS UMR7284, UCA, CHU de Nice

"We generated a new mouse model as part of the "Mouse models and rare diseases" call for projects, launched by the FMR in 2015. This model allowed us to subsequently obtain ANR funding for an ambitious project aimed at understanding how mitochondrial dysfunction can trigger a motor neuron disease, such as amyotrophic lateral sclerosis. We have identified a repositionable molecule, which corrects the defects observed in cultured patient cells, with which we will start a preclinical trial on the mouse model generated through FMR."

Pr Jacques Beckmann

Genetics and Clinical Bioinformatics, Swiss Institute of Bioinformatics, University of Lausanne (Switzerland)

"I am privileged and proud to have been involved with the Rare Diseases Foundation as a member and then Chairman of its Scientific Council for several years. I have seen it come into being, grow, and expand its activities - from research to the promotion of education and communication, then to the creation of links with the world of biotech and logistical support for other foundations - until it assumed an essential role at the European level. I congratulate and thank all those who have contributed to transforming this young initiative into a central player for families and individuals affected by these so-called 'rare' diseases that burden so many people."

Pr Yves Pirson

Professor Emeritus of the Faculty of Medicine of the UCLouvain, Belgium

"I am delighted that the Foundation - whose beginnings and rise to prominence I knew - has now become a key player at the European level.
As a major partner of the European Joint Programme on Rare Diseases, the Foundation is now involved in research efforts aimed at elucidating the mechanisms, accelerating diagnosis and optimizing the clinical and societal management of these diseases, in particular through the management of projects aimed at improving the care pathway and the daily life of patients.

Long live the Foundation and best wishes for success!"

Ms. Nathalie Coulon

President of the IRCEM Foundation

"The Ircem Corporate Foundation was created in 2013 to support actions that enable better living within the family at all ages of life. For 7 years, it has been committed alongside the Rare Diseases Foundation to improve the care, support and quality of life of children and adolescents with rare diseases and their families. Since 2015, she has been convinced of the added value of the Rare Disease Foundation's original positioning in the rare disease ecosystem. Indeed, the Foundation's close accompaniment of field actors likely to be involved in projects and the quality of exchanges between the two foundations have not only allowed research in the human and social sciences to emerge and be promoted, but also to encourage collaboration and co-innovation by involving university researchers, patient associations, and health professionals. Thanks to this solid partnership, the Ircem Corporate Foundation has been able to congratulate itself for contributing to the development of projects "for and with the people concerned" oriented towards the search for solutions (therapeutic, digital...) or social innovations."

Pr Hélène Dollfus

PU-PH, University Hospitals of Strasbourg/INSERM UMRS_1112, Coordinator of the SENSGENE network and the ERN-EYE network

"Thanks to the Rare Diseases Foundation, many research projects have been carried out in the field of rare sensory diseases: from the resolution of diagnostic deadlocks with the revolution of genome sequencing and OMiCs to the human and social sciences, always with the aim of serving the patient community. We are very grateful for this loyal support!

The Foundation also organizes many rich events in which we have been able to participate as partners and we look forward to continuing to do so in the future. These events are essential to bring together all rare disease stakeholders!"

Ms. Marie-Pierre Bichet

President of the Alliance Maladies Rares

"What a pleasure it is to celebrate the 10th anniversary of the Rare Diseases Foundation! The Alliance is proud to be a founding member of this indispensable Foundation for rare diseases.

In 10 years, many projects have emerged for the benefit of patients, families and their associations. We can note, for example, the enormous amount of work undertaken by the Foundation's teams to develop AAPs in Human and Social Sciences, a fundamental field in rare diseases, in order to characterize the disruptions in patients' life paths, specific to the rarity of the diseases.

We are also very pleased with the support offered by the Foundation to rare disease associations that wish to develop their research strategy. The Foundation intervenes in the Alliance congress, in our web conferences and in our autumn universities. Together, we build and update tools that allow patient organizations to become real actors in research (update of the Alliance's research guide). Working together is essential and with the Foundation, it is a reality!

Ms. Catherine Raynaud

Director of Public Affairs, Pfizer France

"The Foundation is an essential link in the world of rare diseases: it has been able to support and create partnerships to support projects which, due to lack of resources, would have had difficulty seeing the light of day, such as research in the human and social sciences, which is fundamental for patients, and, more generally, it plays a major role in supporting all the players on a daily basis.

Mr. Jean-Yves Jaffray

President Kiwanis France-Entraide Foundation

"For more than 100 years, in application of its motto "Serving the children of the World" Kiwanis has mainly provided help to children and families in difficulty: illness, disability, social problems...
But having become aware of the importance of Prevention in the field of illness or disability, we signed a sponsorship agreement between Kiwanis and FMR (April 2017). Many Kiwanian clubs, anonymous donors have mobilized and in five years, €160,000 have been donated to Fundamental Research. And we intend to continue!"

Pr Damien Sanlaville

University Professor, Hospital Practitioner, Head of the Genetics Department, Hospices Civils de Lyon

"The Rare Diseases Foundation has deployed regional managers. Estelle Chanudet, the first regional manager for the Auvergne-Rhône Alpes region, has made it possible to create a link between different academic and private players in the region, creating a breeding ground for future collaborations that have served me well in the construction of the AURAGEN project.

Similarly, the Fondation Maladie Rares has launched calls for GenOmics projects. In 2013, we were able to obtain funding for a whole genome sequencing project. This project allowed us to validate a proof of concept concerning the feasibility of cloning chromosomal breakpoints by whole genome analysis. We were thus able to obtain other funding in this area and to promote our work in international journals.

Pr Laurence Faivre

Genetic Center and Reference Center for Developmental Anomalies and Malformative Syndromes - FHU TRANSLAD, INSERM UMR1231 - Dijon University Hospital

"As a clinical geneticist and researcher, the Foundation has been able to identify funding needs in relation to innovation, given its proximity to the field. The early implementation of funding for new generation sequencing has enabled many French researchers to identify the molecular basis of their pathologies of interest. But I would particularly like to highlight the call for projects in the humanities and social sciences, which has led to some very good multidisciplinary collaborations, and has led to the emergence of very good quality research to identify the needs of patients.

Mr. Antoine Ferry

President of the C.t.r.s. Laboratory

"With the creation of the POC Club, the Foundation For Rare Diseases has certainly made it possible to better promote academic research in this field and, above all, to greatly facilitate exchanges with research teams. The Foundation's investments in the format of presentations, the interventions of researchers and the follow-up to POC meetings are to be commended and allow us, as Pharmaceutical Companies, to envisage partnerships under the best conditions.

I particularly want to thank them because we were able to sign, thanks to the Foundation, a co-maturation agreement for a very relevant project in an extremely rare and particularly serious disease."

Mr. Bruno Caron

President of Mgefi

"Mgéfi has always been committed to the values of mutual aid, support and solidarity shared with the Foundation For Rare Diseases, and which are the source of their strength. This is why, for several years now, we have been proud to support the foundation and its actions focused on "the other", to advance research and help people in pain, as well as their caregivers."

Dr. Paola DE CARLI

Scientific Director of Vaincre la Mucoviscidose

"Over the years, thanks to its team of committed, available and highly competent professionals, the Rare Diseases Foundation has become a key player in supporting research. For Vaincre la Mucoviscidose, its action close to the field and always involving the "winning trio": researcher - caregiver - patient, has been inspiring. Our regular exchanges and the partnership we have built, in the framework of our respective calls for projects in the humanities and social sciences, bear witness to this fruitful and synergistic collaboration."