Rare diseases / Projects

Second Programme of Community action in the Field of Health 2008-2013
UNEW_FY2013 [UNEW_FY2013]
TREAT-NMD is an international network for rare inherited neuromuscular disorders (NMDs). Its mission is to provide an infrastructure to increase collaboration between clinicians, scientists, and patie...
TREAT-NMD is an international network for rare inherited neuromuscular disorders (NMDs). Its mission is to provide an infrastructure to increase collaboration between clinicians, scientists, and patients, accelerate therapy development for these incurable conditions, improve patient care through publication and dissemination of best-practice consensus guidelines, and provide reliable information for patients and professionals. TREAT-NMD was established in 2007, and until the end of 2011, was funded through FP6 to bring together stakeholders representing all NMDs (>200 diseases). Although NMDs are individually rare, TREAT-NMD activities address ~200,000 patients in the EU, and ~3 million worldwide.
Since 2007 TREAT-NMD has increased the collaborating capacity of the field, raised the profile of NMDs within the wider rare disease (RD) field, become a model for RD collaboration, and increased the profile of European NMD research and healthcare globally. A public consultation in 2010 strongly endorsed the continuation and further development of TREAT-NMD`s core activities.
The TREAT-NMD Alliance has extended beyond the original network members to integrate new participants from around the world. The original number of ~350 scientists and clinicians that were integrated into the network through the 22 partners has now more than doubled and constitutes a critical mass for innovation and harmonisation in NMD care and research globally.
TREAT-NMD outputs:
-highly developed communication/dissemination infrastructure with extensive website and newsletter
-network of over 270 NMD care and trial sites
-global patient registries for several NMDs with >20,000 patients
-range of preclinical resources
-tools supporting translational & clinical research
TREAT-NMD vision:
-enhance European excellence in the NMD field
-increase visibility & recognition of NMDs in national health policy
-sustain core resources and added value of networking developing tools that cannot be maintained by individual research projects
-extend networking activities and resources to new experts worldwide
Start date: 01/01/2013 - End date: 01/01/2014

Call: Generate And Disseminate Health Information And Knowledge (Hi-2012)
Topic: Rare diseases
Second Programme of Community action in the Field of Health 2008-2013
EMSP_FY2013 [EMSP_FY2013]
Founded in 1989, EMSP is the umbrella organisation representing 38 national MS societies from 34 European countries. EMSP represents their interests at European level and works collectively with its m...
Founded in 1989, EMSP is the umbrella organisation representing 38 national MS societies from 34 European countries. EMSP represents their interests at European level and works collectively with its members to achieve our goals of high quality equitable treatment and support for Persons with Multiple Sclerosis (PwMS) throughout Europe.

EMSP vision is to move from high quality treatment and support for all PwMS in Europe to a world without MS!

EMSP`s mission is to ensure the development and the implementation of high quality standards of treatment and support for PwMS, their families and their carers in Europe, to allow them to lead their life independently and to recognise them as equal members of the society.

EMSP work priorities to achieve this mission are to:
- influence EU institutions and other decision-making bodies to improve their quality of life for PwMS;
- network with other European organisations which share of vision of truly patient centred approach;
- provide MS member organisations with information on relevant EU draft legislation and how we can influence it, and evidence - based projects that will contribute to their work;
- develop and promote recommendations for improved medical treatment and rehabilitation to be implemented in the EU member states and other countries;
- encourage coordinated research aiming to benefit of PwMS;
- initiate and implement cross-border projects to improve the living conditions of PwMS (e.g. in the field of therapies, employment, social security, infrastructure, etc.);
- act as an information platform by exchanging knowledge and best practices relevant to the work of the national MS member organisations;
- enhance a general awareness on MS by informing and educating external audiences;
- and to support national MS Societies, both recent and long-established, in Europe.
Start date: 01/01/2013 - End date: 01/01/2014

Call: Generate And Disseminate Health Information And Knowledge (Hi-2012)
Topic: Rare diseases
Second Programme of Community action in the Field of Health 2008-2013
EURORDIS_FY2013 [EURORDIS_FY2013]
EURORDIS is a patient-driven alliance of Patient Organisations(POs) and individuals active in the field of rare diseases (RD). It represents the voice of approximately 30 million citizens in the EU.

...
EURORDIS is a patient-driven alliance of Patient Organisations(POs) and individuals active in the field of rare diseases (RD). It represents the voice of approximately 30 million citizens in the EU.

EURORDIS mission is to build a strong pan-European community of POs and People Living With RD (PLWRD), to be their voice at the European level and to fight against the impact of RD on their lives.

To this end, EURORDIS undertakes activities on behalf of its members:

- Empowering RD patient groups
- Advocating RD as a public health priority
- Raising public awareness on RD
- Improving access to information, treatment, care and support for PLWRD
- Improving quality of life
- Networking activities
- Capacity building
- Encouraging good practices in relation to these issues
- Promoting scientific and clinical research on RD
- Promoting the development of treatments and drugs for people living with RD

EURORDIS plays a pivotal role in the implementation of the EU strategy on RD: participation to the EU Committee of Experts on RD (EUCERD) and related road map,elaboration of National Plans or Strategies on RD, European Commission Communication and Council Recommendations in the field of rare diseases, Cross Border Health Care (CBHC) and patient`s mobility, as well as in the implementation of EU Regulations on Orphan Drugs, Paediatrics and Advanced Therapies Medicinal Products (ATMPs) through participation in the EMA Scientific Committees.
Start date: 01/01/2013 - End date: 01/01/2014

Call: Generate And Disseminate Health Information And Knowledge (Hi-2012)
Topic: Rare diseases
Second Programme of Community action in the Field of Health 2008-2013
ESPNreg_FY2013 [ESPNreg_FY2013]
Every year about 9 children per million children develop end-stage kidney disease (ESKD) and require dialysis or a kidney transplant. ESKD is a rare condition, and its treatment is complex and expensi...
Every year about 9 children per million children develop end-stage kidney disease (ESKD) and require dialysis or a kidney transplant. ESKD is a rare condition, and its treatment is complex and expensive. Low patient numbers per country complicate clinical epidemiological research and hamper the development of evidence-based guidelines. This results in large variation of treatment practices and outcomes across Europe. Therefore, the European Society for Paediatric Nephrology (ESPN) and the European Renal Association and European Dialysis and Transplantation Association (ERA-EDTA) have initiated the ESPN/ERA-EDTA Registry. This specialized network collects prospective data on all European children on dialysis or with a kidney transplant.

The Academic Medical Center (AMC) in Amsterdam, the Netherlands, one of the world`s leading medical centres, hosts the ESPN/ERA-EDTA registry and takes care of its financial management.

The ESPN/ERA-EDTA Registry MISSION is to provide high-quality information on all European children on dialysis or with a kidney transplant.
To achieve this mission, we pursue the following activities:
(1) Collect standardized data on children on dialysis or with a kidney transplant from 35 European countries on a yearly basis;
(2) Regularly publish demographic information on paediatric ESKD;
(3) Perform research projects focusing on
- Transnational differences in paediatric ESKD treatment, aiming at reducing health inequalities
- Rare diseases leading to ESKD in children
- Defining optimal strategies to harmonize and reduce practice variation in ESKD management
The demographic information and research results are disseminated online, emailed to all members and contributors, published in high-quality medical journals and presented at national and international congresses.
Covering a population of 630 million in 35 European countries, the ESPN/ERA-EDTA registry is the world`s largest population-based registry for children with ESKD. Our VISION is to provide access to high quality, evidence-based clinical care to all European children.
Start date: 01/01/2013 - End date: 01/01/2014

Call: Promote Health (Hp-2012)
Topic: Rare diseases
Second Programme of Community action in the Field of Health 2008-2013
NEW e-HEALTH SERVICES FOR THE EUROPEAN REFERENCE NETWORK ON RARE ANAEMIAS [e-ENERCA]
The general objective of e-ENERCA is to provide professionals and patients with e-Health tools to assure the same level of access to rare anaemias (RA) services across Europe. e-Health services will b...
The general objective of e-ENERCA is to provide professionals and patients with e-Health tools to assure the same level of access to rare anaemias (RA) services across Europe. e-Health services will be developed through the implementation of three e-platforms endorsed on the ENERCA website. The specific objectives are: 1) to enhance the creation and use of European inventories to gather updated and reliable data on centres of expertise (CEs) and epidemiological figures 2)To facilitate the continuing medical education and best practices 3) To create a telemedicine platform for the provision of health care and medical advice avoiding the need of physical travelling 4) To promote the recognition of CEs on RA by national health authorities and ENERCA as the European Reference Network for RA 5) To develop e-Health tools in the field of RA and promote their use among the scientific community and patients` associations and 6) to assure the project sustainability in the future.
Start date: 01/01/2013 - End date: 01/01/2016

Call: Promote Health (Hp-2012)
Topic: Rare diseases