The TREAT-NMD duchenne muscular dystrophy registries: Conception, design, and utilization by industry and academia

Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216224%3A14110%2F13%3A00069941" target="_blank" >RIV/00216224:14110/13:00069941 - isvavai.cz</a>
Result on the web
<a href="http://dx.doi.org/10.1002/humu.22390" target="_blank" >http://dx.doi.org/10.1002/humu.22390</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1002/humu.22390" target="_blank" >10.1002/humu.22390</a>

Result language
angličtina
Original language name
The TREAT-NMD duchenne muscular dystrophy registries: Conception, design, and utilization by industry and academia
Original language description
Duchenne muscular dystrophy (DMD) is an X-linked genetic disease, caused by the absence of the dystrophin protein. Although many novel therapies are under development for DMD, there is currently no cure and affected individuals are often confined to a wheelchair by their teens and die in their twenties/thirties. DMD is a rare disease (prevalence <5/10,000). Even the largest countries do not have enough affected patients to rigorously assess novel therapies, unravel genetic complexities, and determinepatient outcomes. TREAT-NMD is a worldwide network for neuromuscular diseases that provides an infrastructure to support the delivery of promising new therapies for patients. The harmonized implementation of national and ultimately global patient registries has been central to the success of TREAT-NMD.
Czech name
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Czech description
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Type
J<sub>x</sub> - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)
CEP classification
FH - Neurology, neuro-surgery, nuero-sciences
OECD FORD branch
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Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Publication year
2013
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

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