SCOPE-DMD

Consortium for Products across Europe in Duchenne Muscular Dystrophy

 Coordinatore UNIVERSITY OF NEWCASTLE UPON TYNE 

 Organization address address: Kensington Terrace 6
city: NEWCASTLE UPON TYNE
postcode: NE1 7RU

contact info
Titolo: Dr.
Nome: Amanda
Cognome: Gregory
Email: send email
Telefono: +44 1912824514
Fax: +44 1912824524

 Nazionalità Coordinatore United Kingdom [UK]
 Totale costo 21˙358˙069 €
 EC contributo 5˙999˙210 €
 Programma FP7-HEALTH
Specific Programme "Cooperation": Health
 Code Call FP7-HEALTH-2013-INNOVATION-2
 Funding Scheme CP-FP
 Anno di inizio 2013
 Periodo (anno-mese-giorno) 2013-06-01   -   2016-05-31

 Partecipanti

# participant  country  role  EC contrib. [€] 
1    UNIVERSITY OF NEWCASTLE UPON TYNE

 Organization address address: Kensington Terrace 6
city: NEWCASTLE UPON TYNE
postcode: NE1 7RU

contact info
Titolo: Dr.
Nome: Amanda
Cognome: Gregory
Email: send email
Telefono: +44 1912824514
Fax: +44 1912824524

UK (NEWCASTLE UPON TYNE) coordinator 1˙022˙094.80
2    BIOMARIN NEDERLAND BV

 Organization address address: J H OORTWEG 21
city: LEIDEN
postcode: 2333 CH

contact info
Titolo: Dr.
Nome: Isabel
Cognome: Ferreira
Email: send email
Telefono: +31 614790205
Fax: +31 71 3322088

NL (LEIDEN) participant 2˙356˙654.20
3    BIOSPRING GESELLSCHAFT FUR BIOTECHNOLOGIE MBH

 Organization address address: ALT-FECHENHEIM 34
city: FRANKFURT AM MAIN
postcode: 60386

contact info
Titolo: Dr.
Nome: Hüseyin
Cognome: Aygün
Email: send email
Telefono: +49 6940894471

DE (FRANKFURT AM MAIN) participant 1˙274˙280.00
4    ASSOCIATION INSTITUT DE MYOLOGIE

 Organization address address: BOULEVARD DE L HOPITAL 47-83
city: PARIS
postcode: 75651

contact info
Titolo: Mrs.
Nome: Christelle
Cognome: Gaultier
Email: send email
Telefono: +33 14216 66 46

FR (PARIS) participant 932˙175.00
5    ACADEMISCH ZIEKENHUIS LEIDEN

 Organization address address: Albinusdreef 2
city: LEIDEN
postcode: 2333 ZA

contact info
Titolo: Prof.
Nome: Jan
Cognome: Verschuuren
Email: send email
Telefono: +31 715262097
Fax: +3171 4258253

NL (LEIDEN) participant 414˙006.00

Mappa


 Word cloud

Esplora la "nuvola delle parole (Word Cloud) per avere un'idea di massima del progetto.

treatment    patients    outcome    clinical    dystrophin    disease    diseases    market    protein    functional    innovative    trials    rare    scope    dmd    nmd   

 Obiettivo del progetto (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.'

Altri progetti dello stesso programma (FP7-HEALTH)

CARDIOGENET (2009)

Definition of a genetic network involved in congenital heart disease

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IMPACT (2011)

IMplementation of quality indicators in PAlliative Care sTudy

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CHEARTED (2009)

Gene- Environment Interactions in Heart Development

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