The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia
Date
2013Author
Roy, Anna
Rodrigues, Miriam
Roxburgh, Richard
Lusakowska, Anna
Oliveira, Jorge
Santos, Rosario
Neagu, Elena
Butoianu, Niculina
Artemieva, Svetlana
Rasic, Vedrana Milic
Posada, Manuel
Palau, Francesc
Lindvall, Bjorn
Bloetzer, Clemens
Karaduman, Ayse
Topaloglu, Haluk
Stringer, Angela
Shatillo, Andriy V.
Martin, Ann S.
Peay, Holly
Flanigan, Kevin M.
Salgado, David
von Rekowski, Brigitta
Lynn, Stephen
Heslop, Emma
Gainotti, Sabina
Taruscio, Domenica
Kirschner, Jan
Verschuuren, Jan
Bushby, Kate
Beroud, Christophe
Lochmueller, Hanns
Oflazer, Piraye
Dai, Yi
Barisic, Nina
Kos, Tea
Brabec, Petr
Rahbek, Jes
Lahdetie, Jaana
Tuffery-Giraud, Sylvie
Claustres, Mireille
Leturcq, France
Ben Yaou, Rabah
Walter, Maggie C.
Schreiber, Olivia
Karcagi, Veronika
Herczegfalvi, Agnes
Viswanathan, Venkatarman
Bayat, Farhad
Sarmiento, Isis de la Caridad Guerrero
Ambrosini, Anna
Inal, Serap
Bladen, Catherine L.
Rafferty, Karen
Straub, Volker
Monges, Soledad
Moresco, Angelica
Dawkins, Hugh
Chamova, Teodora
Guergueltcheva, Velina
Korngut, Lawrence
Campbell, Craig
Kimura, En
Ceradini, Francesca
van den Bergen, Janneke C.
Metadata
Show full item recordAbstract
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.
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