The Dutch Dystrophinopathy Database: A National Registry with Standardized Patient and Clinician Reported Real-World Data.
van de Velde NM, Krom YD, Bongers J, Hoek RJA, Ikelaar NA, van der Holst M, Naarding KJ, van den Bergen JC, Vroom E, Horemans A, Hendriksen JGM, de Groot IJM, Houwen-van Opstal SLS, Verschuuren JJGM, van Duyvenvoorde HA, Snijder RR, Niks EH.
van de Velde NM, et al. Among authors: hendriksen jgm.
J Neuromuscul Dis. 2024;11(5):1095-1109. doi: 10.3233/JND-240061.
J Neuromuscul Dis. 2024.
PMID: 39031379
Free PMC article.
BACKGROUND: Duchenne and Becker muscular dystrophy lack curative treatments. Registers can facilitate therapy development, serving as a platform to study epidemiology, assess clinical trial feasibility, identify eligible candidates, collect real-world data, perform post-ma …
BACKGROUND: Duchenne and Becker muscular dystrophy lack curative treatments. Registers can facilitate therapy development, serving as …