European Network for Rare Paediatric Neurological Diseases (nEUroped)

Why are Centres of expertise and European Networks of Reference important? The new project European Network of Reference for Rare Paediatric Neurological Diseases (nEUroped) will give the answer and illustrate the usefulness of this approach. The overall objective of this new 3-year initiative is to develop a network of communication and information sharing across the field of rare nervous system disorders in children characterised by paroxysmal attacks.

nEUroped Centres of reference | centres de référence | centros de referencia | centri di riferimento | centros de referência | Referenz-Netzwerkewill focus on a number of these conditions, amongst which Alternating Hemiplegia of Childhood (AHC), Narcolepsy and Rare Surgically Treatable Epileptic Syndromes (RSTES), that include Cerebellar hamartomas, Hypothalamic hamartomas, Landau-Kleffner syndrome, Nodular heterotopias, Sturge-Weber syndrome, Tuberous Sclerosis and Rasmussen syndrome.

'nEUroped provides a unique opportunity to implement a pilot European Network of Reference, especially at a time when we are promoting the development of this type of networks for rare diseases in national and European policy agendas' says Yann le Cam, Chief Executive Officer of Eurordis. 'nEUroped is a model of partnerships between patients and physicians to create more centres of expertise and tools within networks of reference at European level. If successful, it will serve as a case study to push more in this direction'.

Jasmine, rare disease atient | malade | paciente | paziente | paciente | PatientAn international team of 13 partners (coming from patient organisations, hospitals, universities, research centres and even private companies) from 9 European countries (from the UK to Slovenia) will work together in order to identify main research, health care and social needs for each of the selected diseases. More specifically, the partners will build European-wide patient registries on AHC, Narcolepsy and RSTES and aim to develop guidelines for early diagnosis and treatments for the selected diseases. The project will also serve to identify specific ethical issues and best practice in this field and spread the network's expertise throughout Europe. nEUroped comes from patient groups' strong conviction that by networking and sharing information about their particular disease they can improve diagnosis and help develop new therapeutic interventions. 'nEUroped is a unique opportunity to complement clinical expertise with patients' real life experience' says Dr. Tsveta Schyns, Coordinator of the project and Secretary General of the European Network for Research on Alternating Hemiplegia (ENRAH).

Eurordis' role is pivotal since it will be responsible for integrating patients and patient organisations into the Network. Amongst the patient-oriented activities, feature the organisation of two workshops for Narcolepsy and AHC and several capacity building workshops for patient group representatives on patient registries and on clinical trials. Eurordis will also launch mailing lists for each selected disease. 'The idea behind the lists is to promote the creation of on-line communities of patients, parents, health professionals and anyone interested in knowing more about a disease, in order to benefit from other people's experience,' explains Anja Helm, Manager of Relations with Patient Organisations at Eurordis.

nEUroped focuses on diseases which affect children from early infancy. Most patients have a better chance of being treated if diagnosed in the first year of life. Unfortunately, due to the fact that they are rare and sporadic many of these cases go undiagnosed until the teenage years. Moreover, they are often symptomatically diagnosed or are mistreated because of poor knowledge of the natural evolution of the disease. nEUroped gives renewed hope of finding new clues into these diseases. What is their genetic code? What is their natural evolution? How many people suffer from these diseases? Answers to these questions will improve clinical practice, diagnosis and treatment.

Nathan, rare disease atient | malade | paciente | paziente | paciente | PatientNevertheless, it is important to underline that nEUroped is not a clinical research activity. The project's major strength is its capacity to tap into already-existing clinical data and bring together people who have an expertise and are determined to bridge the gap between patients and scientific research.'Although the aim of nEUroped is above all to provide information to patients and their families, it is also going to support researchers and help them direct their work,' explains Tsveta Schyns, 'The information they will be able to access will be especially valuable because it will be made up of confirmed diagnosis and completed data files only.' At present, research focusing on children is scarce because of the difficulties obtaining ethical approvals and of pursuing follow-up work. 'Most information about paediatric rare neurological diseases is based on case studies,' explains Tsveta Schyns, 'Networking is the only way to access a number of cases that is statistically significant'.

For more information:
contact Tsveta Schyns at ENRAH
 


This article was previously published in the February 2008 issue of our newsletter.


Author: Paloma Tejada
Photos: Centres of reference © Eurordis/B.Ferrier, Jasmine © Anne Carr, Nathan © Dorothea Belmans

 

Page created: 19/08/2009
Page last updated: 20/04/2010
 
 
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