The Foundation For Rare Diseases 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 progress made and to associate all the actors with whom we work every day: researchers, patient associations, rare disease networks and structures, drug manufacturers, and also all our partners and donors who have placed their trust in us!

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

A huge thank you to all

You will find below testimonies from our daily partners.

Ms. Laurence Tiennot-Herment

President of AFM-Telethon

"For the past 10 years, the Rare Diseases Foundation has been able to structure its actions in order 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 National Assembly study group on rare diseases Deputy of 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 financing 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 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 of Nice

"We generated a new mouse model in the framework of the call for projects "Mouse models and rare diseases", launched by the FFRD 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 FFRD."


Pr Jacques Beckmann

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

"I have had the privilege and pride of accompanying the Foundation For Rare Diseases as a member and then president of its scientific council for several years. I have seen it being born, growing, expanding 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 to other foundations - until assuming 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 Hélène Dollfus

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

"Thanks to the Foundation Fore Rare Diseases, many research projects in the field of rare sensory diseases have been initiated: 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 better 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 all rare disease stakeholders together!" 





Ms. Marie-Pierre Bichet

President of the Rare Diseases Alliance

"What a pleasure to celebrate the 10th anniversary of the Foundation For Rare Diseases! The Alliance is proud to be a founding member of this Foundation, which is essential 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 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 who wish to develop their research strategy. The Foundation takes part in the Alliance's congress, in our web conferences, and in our fall 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 a lack of resources, would have found it difficult to see 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 world's children", Kiwanis has mainly provided assistance 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 FFRD (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 Foundation For Rare Diseases has deployed regional managers. Estelle Chanudet, the first regional manager for the Auvergne Rhône-Alpes region, has made it possible to create links between different academic and private players in the region, creating a breeding ground for future collaborations which have served me well in the construction of the AURAGEN project.

Similarly, the FFRD 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. Thus, we were able to obtain other funding on this theme and promote our work in international journals."

Pr Laurence Faivre

Genetics Center and Reference Center for Developmental Anomalies and Malformative Syndromes - FHU TRANSLAD, INSERM UMR1231 - CHU of Dijon

"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 enabled some very good multidisciplinary collaborations, and has led to the emergence of very good quality research to point to needs for patients."

 

 

Mr. Antoine Ferry

President of Laboratoire C.t.r.s.

"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 facilitate exchanges with the 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 would particularly like 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 Mgéfi

"Mgéfi has always been committed to the values of mutual aid, support and solidarity shared with the Foundation For Rare Diseases, and which are their strength. This is why, for several years, we have been proud to support the foundation and its actions turned towards "the other", to advance research and help people in suffering, as much 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 Foundation For Rare Diseases 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 context of our respective calls for projects in the humanities and social sciences, bear witness to this fruitful and synergistic collaboration."