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The rapidly expanding knowledge of NMDs genetic diagnosis, pathogenesis and therapeutic possibilities has provided new targets for disease characterisation, early diagnosis, drug discovery and development as well as has raised many questions about how to translate this knowledge into clinical practice as (initial) clinical trials typically run for such a short time that clinical improvement can hardly be expected within that time frame. This militates for the discovery of surrogate endpoints for establishing the efficacy of clinical trials.

The concept of biomarkers represents measurable bio-parameters able to flank the process of diagnosis, functional characterisation and therapy in NMDs. OMIC sciences (genomic, transcriptomics, proteomics) offer opportunities to identify biomarkers for finely defining and tuning the NMDs bases. This approach can make available non-invasive biomarkers, to be used for monitoring disease progression, prognosis and drugs response, therefore optimising the choice of appropriate and often personalised therapies. Validated biomarkers will increase therapy efficiency (meaning optimal dose of drug to get responders) and efficacy (responders vs non responders for example if we will identify genomic biomarkers linked to the lack of any therapeutic effect).


From 2009-12-01 to 2012-11-30

Project details

Total cost: EUR 7 539 671
EU contribution: EUR 5 634 356
Coordinated in: Italy
Subprogramme: HEALTH-2009-2.4.4-1 - Rare neurological diseases
Call for proposal: FP7-HEALTH-2009-single-stage
Funding scheme: CP-FP - Small or medium-scale focused research project


Coordinator: - UNIVERSITA DEGLI STUDI DI FERRARA - Italy

Participants

ACADEMISCH ZIEKENHUIS LEIDEN - LEIDS UNIVERSITAIR MEDISCH CENTRUM - Netherlands
UNIVERSITY OF NEWCASTLE UPON TYNE - United Kingdom
UNIVERSITA DEGLI STUDI DI PADOVA -Italy
UNIVERSITY COLLEGE LONDON - United Kingdom
UNIVERSITA DEGLI STUDI DI ROMA TOR VERGATAItaly
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) - France
UNIVERSITA DEGLI STUDI DI MILANO - Italy
KUNGLIGA TEKNISKA HOEGSKOLAN - Sweden
NOVAMEN SAS - France
UNIVERSITE D'AIX MARSEILLE - France
ARIADNE DIAGNOSTICS LLC - United States
STEINBEIS GMBH & CO. KG FUER TECHNOLOGIETRANSFER - Germany
ARIADNE GENOMICS INC - United States
LIFE TECHNOLOGIES GMBH - Germany