With determination and hope, Eurordis’ members keep raising the standard of research higher and higher. This will be visible throughout 2007:
• Annual Membership Meeting 2007 in Paris on 4 and 5 May, centred on the topic of access to research resources, at the Pasteur Institute, to celebrate the 10 year anniversary of Eurordis
• Position papers to assert our research priorities
• Development of training sessions for patients in France, Italy, Spain and Denmark
• Development of new research projects to ensure the implementation of a proper research framework in the future.
 

People affected by rare diseases have developed a thorough, intimate and unique knowledge, based on their personal and collective experience of the disease. They are experts in their disease. They are also the initiators, the catalysts and the partners of fundamental and clinical research on their disease. They are true actors in the health system and in research. They are the co-builders of scientific and medical knowledge, along scientists and health professionals. This extraordinary and irreplaceable potential should be recognised and supported, by reinforcing the independent capacity of action of patient representatives, and by building a common action.

To make scientific and medical knowledge their own is an inalienable right of patients. Beyond this right, the understanding of the concepts, vocabulary, policies and instruments of research is at the centre of Eurordis’ current strategic approach. To speak a common language - patients, professionals, and decision makers – and to build a common agenda for all stakeholders of the 7,000 rare diseases and all the countries of Europe, are the political utopia which underlies the Community actions led by Eurordis to serve public interest. More united, more qualified and stronger, we will be more effective in improving our live conditions and those of our loved ones. To ensure that our suffering and our experiences - in which our strength and common vision take root - help all rare (and not so rare) diseases... Best wishes for the holiday season.

Yann Le Cam
Chief Executive Officer

For rare disease patients, who sometimes lack appropriate medical services or treatment in their own country, the issue of patient mobility across European borders is significant. Eurordis has been advocating for patient mobility for the past two years: it is closely linked to our ongoing policy development on centres of expertise for rare diseases in Europe. The opportunity to express our needs and wishes on patient mobility is now open, with the European Commission’s public consultation on health services and patient mobility.

Eurordis is launching a new EU-funded project entitled Capacity Building for Patient Organisations in Research Activities, aiming at giving patients and patient groups the language and tools they need to increase their involvement in clinical trials and better understand EU research policies. Read the article to find out how this project could help you to get involved in research activities on your own disease.

Alliance Sanfilippo is the first international organisation exclusively dedicated to Sanfilippo syndrome. It places high hopes on research and wants to accelerate the pace of biomedical research in the disease. It has been very active since its creation at the end of 2005 and is starting to have an impact on the community.

Jeans for Genes is a UK one-day national appeal that was set up to raise funds for children with genetic diseases. The idea is simple and fun: everyone is invited to throw out the usual dress code, jump into their jeans and show they care by donating £1 or more. This fun charity has raised up to £24 million over a ten year period.

Treatment of Graft-versus-Host disease
Budesonide (oral use)

Treatment of gastric cancer
Catumaxomab

Prevention of rejection of corneal transplant
Ciclosporin (implant)

New designations October 06

Treatment of Li Fraumeni Syndrome
Adenoviral vector containing human p53 gene

Treatment of neonatal brain injury
5-(2,6-Difluoro-phenoxy)-3(R,S)-{2(S)-[2(S)-(3- methoxycarbonyl-2(S)-{3-methyl-2(S)-[(quinoline-2- carbonyl)-amino]-butyrylamino}-propionylamino)-3- methyl-butyrylamino]-propionylamino}-4-oxo- pentanoic acid methyl ester

Treatment of renal cell carcinoma
Genetically modified allogeneic (human) tumour cells for the expression of IL-7, GM-CSF, CD80 and CD154, in fixed combination with a DNA-based double stem loop immunomodulator (dSLIM)

Treatment of renal cell carcinoma
Human Interleukin-2 (glycosylated tetrasaccharide, glycosylated trisaccharide and non-glycosylated) (inhalation use)

Treatment of amyotrophic lateral sclerosis
Arimoclomol

Treatment of glioma
Iodine (131I) anti-tenascin monoclonal antibody 81C6

Treatment of pancreatic cancer
Paclitaxel (liposomal)

All Orphan Drugs in Europe (in English) >

European Public Assessment Reports (EPARs)(multilingual) >

Editorial Team: Yann Le Cam, Jérôme Parisse-Brassens (Editor and Writer), Julia Fitzgerald, Nathacha Appanah, David Oziel (Site of the Month), Anja Helm, François Houÿez, Flaminia Macchia

Translation Team: Conchi Casas Jorde (Spanish), Ana Cláudia Jorge and Victor Ferreira (Portuguese), Roberta Ruotolo (Italian), Trado Verso (French), Ulrich Langenbeck (German)

© 2006 Eurordis