The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia
ABSTRACT 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 th...
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Published in | Human mutation Vol. 34; no. 11; pp. 1449 - 1457 |
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Main Authors | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
Format | Journal Article |
Language | English |
Published |
United States
Blackwell Publishing Ltd
01.11.2013
Hindawi Limited Wiley |
Subjects | |
Online Access | Get full text |
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Summary: | ABSTRACT
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 determine patient 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. For the DMD registries within TREAT‐NMD, individual countries have chosen to collect patient information in the form of standardized patient registries to increase the overall patient population on which clinical outcomes and new technologies can be assessed. The registries comprise more than 13,500 patients from 31 different countries. Here, we describe how the TREAT‐NMD national patient registries for DMD were established. We look at their continued growth and assess how successful they have been at fostering collaboration between academia, patient organizations, and industry.
TREAT‐NMD is a worldwide network for neuromuscular diseases that provides an infrastructure to support the delivery of promising new therapies for patients. |
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Bibliography: | istex:ED09C20547A46A07FCD5E3437FB6E3F693AC792D European Commission and TREAT-NMD - No. FP6 LSHM-CT-2006-036825; No. 20123307 UNEW_FY2013 AFM (Association Francais contre les Myopathies) - No. 16104 ArticleID:HUMU22390 ark:/67375/WNG-G9JFW87H-Q Contract grant sponsor: European Commission and TREAT‐NMD (FP6 LSHM‐CT‐2006–036825, 20123307 UNEW_FY2013, AFM (Association Francais contre les Myopathies) 16104). Communicated by Mark H. Paalman ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 |
ISSN: | 1059-7794 1098-1004 1098-1004 |
DOI: | 10.1002/humu.22390 |