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BIOIMAGE-Neuromuscular Diseases

Objective

BIOIMAGE-NMD will develop and apply imaging technology to monitor response to novel therapies in neuromuscular diseases (NMD) and will use Duchenne muscular dystrophy (DMD) as an exemplar disease. DMD is well characterised genetically and clinically but to date a disease modifying treatment is not available. One of the most promising developments for future treatment of NMD is RNA modulation through antisense oligonucleotides (AON). In DMD, AON are used for exon skipping and this is a genuine example of personalised medicine, where patients are treated according to their specific gene mutation.
Quantitative Magnetic Resonance Imaging (MRI) and Magnetic Resonance Spectroscopic Imaging (MRSI) are used to assess structural and metabolic muscle pathology in NMD but their effectiveness in monitoring therapy is yet to be shown. The project will apply a simultaneous MRI/MRSI protocol in multi-centre clinical trials of AON therapy in DMD with the aim of establishing a clinical proof of principle that these imaging measures are effective biomarkers of therapeutic response. To enhance the imaging protocol, novel Diffusion Tensor MRI (DTI) methods will be developed and optimised to assess muscle microstructure and applied in these trials. The project will also develop methods for radiolabelling of AON and demonstrate the use of pre-clinical Positron Emission Tomography (PET)/MRI to assess the tissue targeting, bio-distribution and pharmacokinetics of AON in vivo.
BIOIMAGE-NMD will deliver PET/MRI and MRI/MRSI technologies for both drug development and clinical evaluation roles which will significantly contribute to bringing personalized therapeutic interventions in rare and common diseases to the market.

Call for proposal

FP7-HEALTH-2013-INNOVATION-1
See other projects for this call

Coordinator

UNIVERSITY OF NEWCASTLE UPON TYNE
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne
United Kingdom
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 1 578 330,41
Administrative Contact
Helen Gallon (Mrs.)

Participants (8)

BIOMARIN NEDERLAND BV
Netherlands
EU contribution
€ 1 216 224,39
Address
J H Oortweg 21
2333 CH Leiden
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Isabel Ferreira (Dr.)
SCITO SA

Participation ended

France
EU contribution
€ 87 572,40
Address
Rue Des Haies 8
75020 Paris
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Arno Klaassen (Dr.)
ASSOCIATION INSTITUT DE MYOLOGIE
France
EU contribution
€ 1 126 177,90
Address
Boulevard De L Hopital 47-83
75651 Paris
Activity type
Research Organisations
Administrative Contact
Christelle Gaultier (Mrs.)
ACADEMISCH ZIEKENHUIS LEIDEN
Netherlands
EU contribution
€ 573 054,93
Address
Albinusdreef 2
2333 ZA Leiden
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Jan Verschuuren (Prof.)
UNIVERSITA CATTOLICA DEL SACRO CUORE
Italy
EU contribution
€ 120 720
Address
Largo Gemelli 1
20123 Milano
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Filippo Leone (Mr.)
KATHOLIEKE UNIVERSITEIT LEUVEN
Belgium
EU contribution
€ 120 720
Address
Oude Markt 13
3000 Leuven
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Elke Lammertyn (Dr.)
UCL Elizabeth Garrett Anderson Institute for Women’s Health
United Kingdom
EU contribution
€ 637 532,56
Address
Gower Street
WC1E 6BT London
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Gosia Kielbasa (Ms.)
CONSULTANTS FOR RESEARCH IN IMAGING AND SPECTROSCOPY SCRL
Belgium
EU contribution
€ 500 200,40
Address
Chaussee De Lannoy 31
7503 Tournai
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Raymond Gilles (Dr.)