The submission of full proposals is now CLOSED!
Attention: there were modification in the document « Guidelines for applicants » concerning Italian and Latvia participants. The updated document is available for download.
Please note that the information provided on E-Rare website is not exhaustive. For all details of the call, eligibility criteria and sumbission rules please download and read documents provided in "Download documents" section.
In order to submit your proposal you need to register to the submission system via: https://www.pt-it.de/ptoutline/ERARE15
The ERA-Net “E-Rare” for research programmes on rare diseases has been extended to a third phase “E-Rare-3” (2014-2019) to further help in coordinating the research efforts of European countries in the field of rare diseases and implement the objectives of International Rare Disease Research Consortium (IRDiRC). The following parties:
intend to open the seventh E-Rare joint call for funding multilateral research projects on rare diseases together with the European Commission (EC) under the ERA-Net co-fund mechanism. The call is expected to be opened simultaneously by the parties in their respective countries.
AIM AND TOPICS
The aim of the call is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with a clear translational research approach.
Subject: a group of rare diseases or a single rare disease following the European definition i.e. a seriously debilitating and/or life-threatening disease affecting not more than five in 10.000 persons in the EU, EC associated states and Canada
Participating countries: Austria, Belgium (Flanders), Belgium (French speaking community), Canada (including Québec), France, Germany, Greece, Hungary, Israel, Italy, Latvia, Poland, Portugal, Romania, Spain, Switzerland, The Netherlands and Turkey.
Topics:
Transnational research proposals must cover at least one of the following areas, which are equal in relevance for this call:
Project proposals must clearly demonstrate the potential health impact as well as the added-value of transnational collaboration: gathering a critical mass of patients/biological material, sharing of resources (models, databases, diagnosis etc.), harmonization of data, sharing of specific know-how and/or innovative technologies, etc.
Each transnational collaborative project should represent the critical mass to achieve ambitious scientific goals.
Consortia are encouraged to demonstrate engagement with industry for its active participation including areas of collaboration, sharing of resources, capabilities and expertise, in order to ensure an efficient transfer of pre-clinical results into clinical utility. Likewise, patient organizations are invited to participate where appropriate as their engagement has the potential to provide new insights that could lead to innovative discoveries, and ensures that research is relevant to patients' concerns.
It is expected that the inclusion of partner groups from participating Eastern European countries will contribute to strengthening the research capacity building in Europe as a whole.
Number of partners requesting funding | Possible number of additional partners with own funding |
3 | 2 |
4 | |
5 | |
6 | |
7 (only possible with inclusion of 1 Eastern European partner) | 1 |
8 (only possible with inclusion of 2 Eastern European partner) | 0 |
The submission of pre-proposals is now CLOSED!
Attention: there were modification in the document « Guidelines for applicants » concerning Italian and Latvia participants. The updated document is available for download.
Please note that the information provided on E-Rare website is not exhaustive. For all details of the call, eligibility criteria and sumbission rules please download and read documents provided in "Download documents" section.
In order to submit your proposal you need to register to the submission system via: https://www.pt-it.de/ptoutline/ERARE15
1. MOTIVATION
There are at least 6.000 to 7.000 distinct rare diseases, the great majority being of genetic origin. Although individually rare, taken together rare diseases affect at least 26-30 million people in Europe. Moreover, they represent a major issue in health care: a large number of these diseases lead to a significant decrease of life expectancy and most of them cause chronic illnesses with a large impact on quality of life and the health care system.
Therefore, research on rare diseases is needed to provide knowledge for prevention, diagnosis and better care of patients. Yet, research is hampered by lack of resources at several levels: (1) Few scientists work on one specific disease, (2) There are few patients per disease and they are scattered over a large geographic area, causing difficulties to gather the necessary cohorts, (3) Existing databases and material collections are usually local, small, and not accessible or standardised, (4) Diseases often have complex clinical phenotypes and require interdisciplinary cooperation for research, hence, interdisciplinary approaches to treatment.
The specificities of rare diseases - limited number of patients, scarcity of relevant knowledge and expertise, and fragmentation of research - single them out as a distinctive domain of very high European added-value. Rare diseases are therefore a prime example of a research area that can strongly benefit from collaboration/coordination on a transnational scale.
In this context, the ERA-Net “E-Rare” for research programmes on rare diseases has been extended to a third phase "E-Rare-3" (2014-2019) to further help in coordinating the research efforts of European countries in the field of rare diseases and implement the objectives of International Rare Diseases research Consortium (IRDiRC)..
The following parties,
have decided to open the seventh E-Rare joint transnational call (JTC 2015) for funding multilateral research projects on rare diseases together with the European Commission (EC) under the ERA-NET Cofund mechanism. The call is being opened simultaneously by the partners in their respective countries.
2. AIM OF THE CALL
The aim of the call is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with a clear translational research approach.
Projects shall involve a group of rare diseases or a single rare disease following the European definition (a disease affecting not more than 5 in 10.000 people in the European Community, EC associated states and Canada).
Transnational research proposals must cover at least one of the following areas, which are equal in relevance for this call:
a) Collaborative research using patient databases and corresponding collections of biological material that would generally not be possible at a national scale. This research must have clear potential for clinical application and well defined scientific objectives e.g.:
• definition of new nosological entities, epidemiological studies, genotype/phenotype correlations, natural history of diseases;
• characterisation of the genetic/molecular basis of specific diseases
b) Research on rare diseases including genetic, epigenetic, and pathophysiological studies, using innovative and shared resources, technologies (OMICS, new generation sequencing, etc.) and expertise.
c) Research on development of applications for diagnosis and therapies for rare diseases. This may include identification and characterisation of (bio)-markers for diagnosis and prognosis, development of innovative screening systems and diagnostic tools, the generation of relevant cellular and/or animal models, and preclinical studies using pharmacological, gene or cell therapies.
Project proposals must clearly demonstrate the potential health impact as well as the added-value of transnational collaboration: gathering a critical mass of patients/biological material, sharing of resources (models, databases, diagnosis etc.), harmonisation of data, sharing of specific know-how and/or innovative technologies, etc.
Each transnational collaborative project should represent the critical mass to achieve ambitious scientific goals. Consortia are encouraged to demonstrate engagement with industry for its active participation including areas of collaboration, sharing of resources, capabilities and expertise, in order to ensure an efficient transfer of pre-clinical results into clinical utility. Likewise, patient organizations are invited to participate where appropriate as their engagement has the potential to provide new insights that could lead to innovative discoveries, and ensures that research is relevant to patients' concerns.
It is expected that the inclusion of partner groups from participating Eastern European countries will contribute to strengthening the research capacity building in Europe as a whole.
The aim of the call is in compliance with the goals set by the International Rare Diseases Research Consortium (IRDiRC) which fosters international collaboration in rare diseases research. For more information see IRDiRC website: http://www.irdirc.org/
The following diseases/topics are excluded from the scope of the call:
• Interventional clinical trials;
• Rare infectious diseases, rare cancers and rare adverse drug events in treatments of common diseases;
• Rare neurodegenerative diseases which are within the focus of the Joint Programming Initiative on Neurodegenerative Disease Research (JPND; http://www.neurodegenerationresearch.eu/). These concern: Alzheimer’s disease (AD) and other dementias; Parkinson’s disease (PD) and PD-related disorders; Prion disease; Motor neuron diseases (MND); Huntington’s Disease (HD); Spinal muscular atrophy (SMA) and dominant forms of Spinocerebellar ataxia (SCA). Interested researchers should refer to the relevant JPND calls.
3. MANAGEMENT BOARDS
Two boards, the Call Steering Committee (CSC) and the Scientific Evaluation Committee (SEC), will manage the evaluation process of the call with support of the Joint Call Secretariat (JCS) (set up at PT-DLR, Germany). SEC and CSC members will not submit or participate in proposals within this call. The process includes the evaluation procedure of pre- and full-proposals and the final selection and award of research projects.
• The Call Steering Committee (CSC) is composed of a single representative from each country/region funding organisation. The CSC will supervise the progress of the call and the evaluation of proposals. The CSC will make the final funding recommendation to the national/regional funding organisations on the proposals to be funded, based on the final ranking list provided by the SEC. All decisions concerning the call procedures will be taken by the CSC. The CSC will ensure that the procedures are implemented in accordance to the ERA-NET Cofund rules.
• The Scientific Evaluation Committee (SEC) is a panel of internationally recognised scientific experts responsible for the evaluation of submitted proposals. SEC members must sign a confidentiality form and a statement to confirm that they do not have any conflicts of interest. The SEC will nominate external peer reviewers.
4. APPLICATION
4.1. Funding recipients/Eligibility
Joint research proposals may be submitted by applicants belonging to one of the following categories (according to country/regional regulations):
• academia (research teams working in universities, other higher education institutions or research institutes)
• clinical/public health sector (research teams working in hospitals/public health and/or other health care settings and health organisations)
• enterprise (all sizes of private companies). Participation of small and medium-size enterprises (SMEs) is encouraged when allowed by national/regional regulations
Please note that the inclusion of a non-eligible partner in a proposal leads to the rejection of the entire proposal without further review. Whilst applications will be submitted jointly by research groups from several countries, individual groups will be funded by the individual funding organisation of their country/region that is participating in the E-Rare-2 JTC 2015. The applications are therefore subject to eligibility criteria of individual funding organisations. Applicants are strongly advised to contact their corresponding national/regional representative and confirm eligibility with their respective funding organisations in advance of submitting an application (see national/regional contact details and Annex). The adherence to the national/regional regulations in the "Guidelines for applicants" document is mandatory.
Only transnational projects will be funded. Each consortium submitting a proposal must involve a minimum of three eligible and a maximum of six eligible partners from at least three different countries participating to the call (see list above). No more than two eligible partners from the same country participating in the call will be accepted in one consortium.
Applicants are encouraged to include partners from the participating Eastern European countries (Hungary, Latvia, Poland, Romania and Turkey). If they include such partners, the maximum number of partners can be increased to seven or eight (see table below).
Additional partners that secure their own funding may join consortia. However, their number is limited to two. The consortium coordinator must always be eligible to receive funding from the funding organisations participating in the call. Only groups that contribute substantially to at least one of the work packages are considered as partners. They must state clearly in the proposal if these funds are already secured or if not, how they plan to obtain funding in advance of the project start. It will be required to document the availability of their funds before October 1, 2015.
Number of partners requesting funding | Possible number of additional partners with own funding |
3 | 2 |
4 | |
5 | |
6 | |
7 (only possible with inclusion of 1 Eastern European partner) | 1 |
8 (only possible with inclusion of 2 Eastern European partners) | 0 |
Each transnational proposal must nominate a project consortium coordinator among the project partner principal investigators. The coordinator must be a project partner from an E-Rare-3 JTC 2015 funding country/region. The project coordinator will represent the consortium externally and towards the JCS and CSC, and will be responsible for its internal scientific management (such as controlling, reporting, intellectual property rights issues and contact with the JCS). Each project partner will be represented by a single principal investigator. Within a joint proposal, the principal investigator of each project partner will be the contact person for the relevant country/regional funding organisation.
Consortia of projects funded in previous E-Rare joint transnational calls can apply for funding for an extension of their cooperation. These consortia must clearly demonstrate the success of the current project and innovative scientific aims for their future collaboration. Their applications will compete with applications for new research projects.
The duration of the projects can be up to 3 years. Nevertheless, a partner can receive funding for less than 3 years according to E-Rare-3 JTC 2015 funding organisations eligibility criteria and regulations.
5.2. Eligibility check of pre-proposals and first step peer review
5.2.1. Eligibility check
The JCS will check all pre-proposals to ensure that they meet the call’s formal criteria (date of submission; number and country distribution of participating research groups; inclusion of all necessary information in English, page length of each section). The JCS will forward the proposals to the CSC members who will perform a check for compliance to country/region rules.
Please note that proposals not meeting the formal criteria or the national/regional eligibility criteria and requirements will be declined without further review.
5.2.2. Peer review of pre-proposals
Proposals passing the eligibility check (call secretariat and country/region) will be forwarded to the SEC members for a first evaluation (see evaluation criteria above). The SEC members will perform the assessment of the pre-proposal and fill the evaluation forms with scores and comments for each criteria. Each pre-proposal will be assessed by 2 SEC members. The SEC members will meet to establish a ranking of the proposals. The CSC will meet to decide which proposals will be accepted for the full proposal submission based on the SEC recommendations.
5.3. Evaluation of full proposals with right to reply (rebuttal stage)
5.3.1. Formal criteria check
The JCS will check the full proposals to ensure that they meet the call’s formal criteria and have not changed substantially from the respective pre-proposals before sending them to the external reviewers.
5.3.2. External reviewer’s evaluation
Each proposal will be allocated to at least two external reviewers who fit the profile of the application.
5.3.3. Rebuttal stage
Before the SEC members see the reviews from external reviewers, each project coordinator will be provided with the opportunity of studying the assessments and commenting on the arguments and evaluations of the reviewers, which remain anonymous. This stage allows applicants to comment on factual errors or misunderstandings that may have been committed by the external reviewers while assessing their proposal and to reply to reviewers’ questions. However, issues which are not related with reviewers’ comments or questions cannot be addressed and the work plan cannot be modified at this stage.
The applicants will have up to one week (between the second and third week of July) for this optional response to the reviewers’ comments.
5.3.4. SEC evaluation
The JCS will send full proposals, reviews and rebuttals to the SEC members. The SEC will meet to discuss each proposal and, after consideration of the evaluation criteria, external reviews, rebuttals and their own discussions, the SEC will make a classification of the proposals and rank proposals recommended for funding.
5.4. Funding decisionBased on the ranking list established by the SEC and on available funding the CSC will suggest the projects to be funded to the national/regional funding organisations. Based on these recommendations, final decisions will be made by the national/regional funding organisations and will be subject to budgetary considerations.
If necessary, the CSC will determine a priority order for proposals which have been awarded the same score within a ranked list. The following approach will be applied successively for every group of ex aequo proposals requiring prioritisation, starting with the highest scored group, and continuing in descending order:
• Availability of national funding;
• Maximization of use of national funding;
• Proposals that address diseases not otherwise covered by more highly-ranked proposals;
• Proposals with participation of Eastern European countries
The joint selection list of projects to be funded will be submitted to the EC together with other relevant information. The Joint Call Secretariat will communicate to all project coordinators the final decisions together with the review from the SEC.
6. FINANCIAL AND LEGAL ISSUES
6.1. Funding model
The E-Rare-3 JTC 2015 Funding Partners have agreed to launch a joint call using the “virtual common pot” funding mode. This means that national/regional funding will be made available through national/regional funding organisations according to national/regional funding regulations. In addition, the EC will also provide funding that will maximize the number of selected projects that can be funded in rank order. Funding from the EC will be distributed through the national/regional funding agencies. Each country/region funds only its national/regional component of the transnational research project. Eligible costs and funding rates may vary according to the corresponding national/regional funding organisation regulations. Prior to submitting a proposal, applicants should verify their eligibility and financial support and are recommended to contact their national/regional contact person (see national/regional contact details).Funding is granted for a maximum of three years according to national/regional regulations.
6.2. Funding contracts
Each project includes several consortium members called research partners and one project coordinator. Each research partner (including the project coordinator) will have a separate funding contract/letter of grant according to national/regional regulations with the appropriate national/regional funding institutions.
Changes to the composition of research consortia or in budget cannot occur during the contract/letter of grant, unless there is a good justification. Any minor changes have to be well justified and the relevant funding organisations will decide upon the proper action to be taken. However, in case of major changes, an independent expert can be consulted to help with the final decision of the funding organisations. The research partners shall inform the JCS and the funding bodies of that project of any event that might affect the implementation of the project.
6.3. Research consortium agreement and ownership of intellectual property rights
The project consortium partners must sign a consortium agreement (CA) for cooperation addressing the issues given in “Guidelines for applicants” on consortium agreements (available on the E-Rare website). The research consortium is strongly encouraged to sign this CA before the official project start date, and in any case the CA has to be signed no later than six months after the official project start date. Please note that national/regional regulations may apply concerning the requirement for a CA (ANR will require the CA to be signed before the start of the project. Please contact your national/regional contact point or check the country-specific information on the guidelines). Upon request, this consortium agreement must be made available to the concerned E-Rare-3 JTC 2015 funding organisations.
Results and new Intellectual Property Rights (IPR) resulting from projects funded through theE-Rare-3 Joint Transnational Call will be owned by the researchers’ organisations according to national/regional rules on IPR. If several participants have jointly carried out work generating new IPR, they shall agree amongst themselves (consortium agreement) as to the allocation of ownership of IPR, taking into account their contributions to the creation of those IPR as well as the European guidelines on IPR issues. The results of the research project and IPR created should be actively exploited and made available for use, whether for commercial gain or not, in order for public benefit to be obtained from the knowledge created. The funding partners shall have the right to use documents, information and results submitted by the research partners and/or to use the information and results for their own purposes, provided that the owner’s rights are kept and taking care to specify their origin.
6.4. IRDiRC policies and guidelines
The project partners are expected to follow IRDiRC policies and guidelines and to participate in IRDiRC working groups. For more information see http://www.irdirc.org/.
7. RESPONSIBILITIES, REPORTING REQUIREMENTS AND DISSEMINATION
The coordinators of all the funded projects must submit brief annual scientific project reports and a final scientific project report (within six months of the end of the project) to FNRS, Belgium, which is responsible for monitoring the funded projects monitoring. All reports must be in English and use a common electronic reporting form that will be provided. The research partners are jointly responsible for delivery of the reports, and FNRS will only accept reports delivered on behalf of the consortium, via the project coordinator.
If required, each participant should submit financial and scientific reports to their national/regional funding organisations, according to national/regional regulations. The progress and final results of each individual contract/letter of grant will be monitored by the respective national/regional funding organisations.
The coordinators and/or national/regional group leaders might be asked to present the results of their projects at an intermediate and/or a final status symposium organized by E-Rare. Project proposal budget should foresee these expenses accordingly.
Funding recipients must ensure that all outcomes (publications, etc.) of transnational E-Rare-3 projects include a proper acknowledgement of ERA-NET E-Rare-3 and the respective national/regional funding partner organisations.
In addition, unless the EC requests or agrees otherwise or unless it is impossible, any dissemination of results (in any form, including electronic) must:
(a) display the EU emblem and
(b) include the following text:
“This project has received funding from the European Union’s Horizon 2020 research and innovation programme under the ERA-NET Cofund action N° 643578”.
When displayed together with another logo, the EU emblem must have appropriate prominence.
For the purposes of the obligations under this Article, the beneficiary may use the EU emblem without first obtaining approval from the Agency.
This does not however give it the right to exclusive use.
Moreover, the beneficiary may not appropriate the EU emblem or any similar trademark or logo, either by registration or by any other means.
8. CONTACT AND FURTHER INFORMATION
The JCS is set up at PT-DLR (The German project management Agency) to assist the CSC and the national/regional funding bodies during the implementation of the call. FNRS, Belgium, will be responsible for the follow-up phase until the funded research projects have ended. The JCS will be responsible for the administrative management of the call. It will be the primary point of contact referring to the call procedures between the research consortia, the funding organisations (CSC) and the peer reviewers. The project coordinator will be the person contacted by the JCS during the application procedure, so he/she must forward this information to the other participants.
Further information on the E-Rare-3 Project, the Call and the follow-up is available at the E-Rare website (www.e-rare.eu). It is advised to contact the national/regional contact person for any questions regarding the Call (please see national/regional contact details below).
INDICATIVE FUNDING COMMITMENT
Country/Region | Participating organisation | Envisioned amount of funding (M€ for 3 years) | Anticipated number of fundable research partners |
Austria | Austrian Science Fund (FWF) | 0.75 | 3 |
Austria | Austrian Research Promotion Agency (FFG) | 2 | 4 |
Belgium | Research Foundation Flanders (FWO) | 0.4 | 2 |
Belgium | Fund for Scientific Research - FNRS (F.R.S.-FNRS) | 0.24 | 1 |
Canada | Canadian Institutes of Health Research Institute of Genetics(CIHR-IG) [1] |
1.06
| 3-5 |
Canada | Fonds de recherche du Québec-Santé (FRQS) | 0.36 | 1-2 |
Canada | Genome Canada (GC) [2] | 0.70* | 2-3 |
France | French National Research Agency (ANR) | 2 | 8-10 |
Germany | German Federal Ministry of Education and Research (BMBF)
German Research Foundation (DFG) | 3
1 | 14-18 |
Greece | General Secretariat for Research and Technology (GSRT) | 0.5 | 5 |
Hungary | Hungarian Scientific Research Fund(OTKA) | 0.15 | 1-2 |
Israel | Chief Scientist Office of the Ministry of Health (CSO/MOH)[3] | 0.24 | 4 |
Italy | Ministry of Health (MoH) | 1.2 | 4-6 |
Italy | Regione Emilia-Romagna - Agenzia Sanitaria e Sociale Regionale (RER-ASSR), Italy | 0.75 | 3-4 |
Latvia | Latvian Academy of Sciences (LAS) | 0.21 | 1-2 |
Poland | National Centre for Research and Development (NCBR), | 0.5 | 3-5 |
Portugal | Foundation for Science and Technology (FCT) | 0.325 | 2-3 |
Romania | Executive Agency for Higher Education, Research, Development& Innovation Funding (UEFISCDI) | 0.5 | 1-6 |
Spain | National Institute of Health Carlos III (ISCIII) | 0.5 | 3-5 |
Switzerland | Swiss National Science Foundation (SNSF)[4] | 0.82 | 2-4 |
The Netherlands | Netherlands Organization for Health Research and Development (ZonMw) | 1.8 | 8-12 |
Turkey | Scientific and Technological Research Council of Turkey (TÜBITAK) | 1 | 8-9 |
[3]The CSO/MOH will fund up to 4 projects. The funding commitment is up to 240,000 €, depending on budget availability. Maximum funding per grant is 60,000 €.
[4] SNSF has earmarked a budget of 1.0 million Swiss Francs over three years (currently equivalent to 0.82 Mio €).
ELIGIBILITY OF BENEFICIARY INSTITUTIONS
Applicants need to contact their national/regional contact points for further information and refer to the national/regional information in the “Guidelines for applicants” document
Country/Region | Institution | Eligible benificiary institution | ||
Academia | Clinical/public health | Company | ||
Austria | Austrian Science Fund (FWF) | Yes (3) | Yes (3) | Yes (3) |
Austria | Austrian Research Promotion Agency (FFG) | No | No | Yes (12) |
Belgium (Flanders) | Research Foundation Flanders (FWO) | Yes | Yes (1,2) | No |
Belgium (french speaking community) | Fund for Scientific Research - FNRS (F.R.S.-FNRS) | Yes (6) | Yes (7,8) | No |
Canada | Canadian Institutes of Health Research Institute of Genetics (CIHR-IG) | Yes | Yes | No |
Canada (Québec) | Fonds de recherche du Québec-Santé (FRQS) | Yes | Yes | No |
Canada | Genome Canada (GC) | Yes | Yes | No |
France | French National Research Agency (ANR) | Yes | Yes | Yes (1) |
Germany | German Federal Ministry of Education and Research (BMBF) | Yes | Yes | Yes (1) |
Germany | German Research Foundation (DFG) | Yes (10) | Yes (11) | No |
Greece | General Secretariat of Research and Technology (GSRT) | Yes | Yes | Yes |
Hungary | Hungarian Scientific Research Fund(OTKA) | Yes | Yes | No |
Israel | Chief Scientist Office of the Ministry of Health (CSO/MOH) | Yes | Yes | No |
Italy | Ministry of Health (MoH) Italy | No | Yes (4) | No |
Italy | Regione Emilia-Romagna - Agenzia Sanitaria e Sociale Regionale (RER-ASSR) | Yes (13) | Yes (13) | No |
Latvia | Latvian Academy of Sciences (LAS) | Yes | Yes | Yes (1) |
Poland | National Centre for Research and Development (NCBR) | Yes | Yes | Yes |
Portugal | Foundation for Science and Technology (FCT) | Yes | Yes | Yes (1) |
Romania | Executive Agency for Higher Education, Research, Development & Innovation Funding (UEFISCDI), | Yes | Yes | Yes (1) |
Spain | National Institute of Health Carlos III (ISCIII), | No | Yes | No |
Switzerland | Swiss National Science Foundation (SNSF) | Yes (9) | Yes (9) | No |
The Netherlands | Netherlands Organization for Health Research and Development (ZonMw) | Yes (5) | Yes (5) | No (5) |
Turkey | Scientific and Technological Research Council of Turkey (TÜBITAK) | Yes | Yes | Yes (1) |
| Year | Acronym | Project Coordinator | Title |
|---|---|---|---|
2015 | Induced pluripotent stem cells for identification of novel drug combinations targeting cystic fibrosis lung and liver disease | ||
2015 | Small Antibody Fragment as Alternative Tools in Haemophilia Care | ||
2015 | European network on Noonan syndrome and related disorders | ||
2015 | Translating Peroxisome Biogenesis Disorders: Identifying Pharmacological Therapies and Clinical Trial Endpoints | ||
2015 | Tracing the untackled facets of Peeling Skin Disease-Targeting epidermal proteolysis for treatment | ||
2015 | Non-SCID combined immunodeficiencies: a diagnostic and therapeutic challenge | ||
2015 | Patient-Derived Glial Precursor Cell Therapy for Vanishing White Matter Disease | ||
2015 | Improving diagnosis and treatment of catecholaminergic polymorphic ventricular tachycardia: integrating clinical and basic science | ||
2015 | Mitochondrial Disorders: from a genome-wide Registry to medical genomics, toward molecular mechanisms and new therapies | ||
2015 | A comprehensive clinical and experimental approach to personalized molecular medicine in patients with defined and undefined autoinflammatory disorders | ||
2015 | A European research network directed towards improving diagnosis and treatment of inborn glycosylation disorders. | ||
2015 | Understanding primary hyperoxaluria type 1 towards the development of innovative therapeutic strategies. | ||
2015 | Netherton Syndrome; From mechanism to therapies | ||
2015 | IMPROVING CARE FOR COHESINOPATHIES: FROM HEART PHENOTYPES TO NOVEL THERAPIES | ||
2015 | Modulation of Neuregulin signaling as an effective strategy to treat hereditary neuropathies (Charcot-Marie-Tooth disease) | ||
2015 | Novel Gene Therapy for Thalassemia: Pre-clinical Development and Assessment in Animal and Stem Cell Models | ||
2015 | Preparing for therapies in autosomal recessive ataxias | ||
2015 | Harmonising phenomics information for a better interoperability in the RD field | ||
2015 | The European Diamond-Blackfan Anemia Consortium |
Looking for collaborations | Contact | Interactive FAQ |
For more information ABOUT E-Rare please contact:
E-Rare Coordinator
Daria JULKOWSKA
Tel.: +33(0) 1 78 09 80 78
daria.julkowska@agencerecherche.fr
Agence Nationale de la Recherche - ANR
Health & Biology Department
50 Avenue Daumesnil
75012 Paris, France
E-Rare 2012 - Created by Toussaint Biger
