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Consortium for Products across Europe in Duchenne Muscular Dystrophy

Objective

"This project builds on results from the TREAT-NMD and BIO-NMD European projects to develop a personalised-medicinal product through to market for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is an inheritable, childhood rare disease that affects approximately 1 in 3,500 newborn boys. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, typically rendering them wheelchair-bound before the age of 12 and most patients die in early adulthood due to respiratory and cardiac failure. At present, there is no effective treatment for DMD. The goal of the SCOPE-DMD project is to develop a therapy that can restore the expression of a functional dystrophin protein in a targeted DMD patient sub-population. This will be achieved using RNA-based technology, specifically antisense oligonucleotides (AON) inducing exon skipping. The clinical heterogeneity and disease complexity in rare diseases present a major challenge to properly observe statistically and clinically meaningful treatment effects. Innovative clinical study designs and novel outcome measures are required to reduce development timelines. Based on extensive experience from classical clinical trials of two other AONs in DMD patients, the consortium has designed a highly innovative development plan, including an innovative clinical trial design that if successful, could be applied to future clinical trials in DMD and other rare diseases. Also novel biomarkers and functional outcome measures will be incorporated in additional to more established ones. Multiple layers of expertise and scientific knowledge come together in this SCOPE-DMD project consortium to launch an innovative drug product but also to provide a regulatory and pathway-to-market precedence in order to benefit future patients with rare disorders getting earlier access to treatment."

Call for proposal

FP7-HEALTH-2013-INNOVATION-2
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

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
€ 1 022 094,80
Administrative Contact
Amanda Gregory (Dr.)

Participants (4)

BIOSPRING GESELLSCHAFT FUR BIOTECHNOLOGIE MBH
Germany
EU contribution
€ 1 274 280
Address
Alt-fechenheim 34
60386 Frankfurt Am Main
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Hüseyin Aygün (Dr.)
ASSOCIATION INSTITUT DE MYOLOGIE
France
EU contribution
€ 932 175
Address
Boulevard De L Hopital 47-83
75651 Paris
Activity type
Research Organisations
Administrative Contact
Christelle Gaultier (Mrs.)
BIOMARIN NEDERLAND BV
Netherlands
EU contribution
€ 2 356 654,20
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.)
ACADEMISCH ZIEKENHUIS LEIDEN
Netherlands
EU contribution
€ 414 006
Address
Albinusdreef 2
2333 ZA Leiden
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Jan Verschuuren (Prof.)