The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study
Craig M. McDonald,Erik K Henricson,R. Ted Abresch,Julaine Florence,Michelle Eagle,Eduard Gappmaier,Allan M. Glanzman,R. Spiegel,Jay A. Barth,Gary Elfring,A. Reha,Stuart W. Peltz +11 more
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TLDR
The 6MWD is an optimal primary endpoint for Duchenne muscular dystrophy clinical trials that are focused therapeutically on preservation of ambulation and slowing of disease progression.Abstract:
Introduction: An international clinical trial enrolled 174 ambulatory males ≥5 years old with nonsense mutation Duchenne muscular dystrophy (nmDMD). Pretreatment data provide insight into reliability, concurrent validity, and minimal clinically important differences (MCIDs) of the 6-minute walk test (6MWT) and other endpoints. Methods: Screening and baseline evaluations included the 6-minute walk distance (6MWD), timed function tests (TFTs), quantitative strength by myometry, the PedsQL, heart rate–determined energy expenditure index, and other exploratory endpoints. Results: The 6MWT proved feasible and reliable in a multicenter context. Concurrent validity with other endpoints was excellent. The MCID for 6MWD was 28.5 and 31.7 meters based on 2 statistical distribution methods. Conclusions: The ratio of MCID to baseline mean is lower for 6MWD than for other endpoints. The 6MWD is an optimal primary endpoint for Duchenne muscular dystrophy (DMD) clinical trials that are focused therapeutically on preservation of ambulation and slowing of disease progression. Muscle Nerve 48: 357–368, 2013read more
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Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy.
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TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy
Pietro Spitali,Irina Zaharieva,Stefan Böhringer,Monika Hiller,Amina Chaouch,Andreas Roos,C. Scotton,Mireille Claustres,Luca Bello,Craig M. McDonald,Eric P. Hoffman,Cinrg Investigators,Zaïda Koeks,H. Eka D. Suchiman,Sebahattin Cirak,Mariacristina Scoto,Mojgan Reza,Peter A C 't Hoen,Erik H. Niks,Sylvie Tuffery-Giraud,Hanns Lochmüller,Alessandra Ferlini,Francesco Muntoni,Annemieke Aartsma-Rus +23 more
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Effect of Combination l-Citrulline and Metformin Treatment on Motor Function in Patients With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Patricia Hafner,Ulrike Bonati,Andrea Klein,Andrea Klein,Andrea Klein,Daniela Rubino,Vanya Gocheva,Simone Schmidt,Simone Schmidt,Jonas Schroeder,G. Bernert,Vincent Laugel,Maja Steinlin,Andrea Capone,Monika Gloor,Oliver Bieri,Lars G. Hemkens,Benjamin Speich,Thomas Zumbrunn,Nuri Gueven,Dirk Fischer,Dirk Fischer +21 more
TL;DR: In this article, the benefits and harms of a combination of l-citrulline and metformin treatment among patients with Duchenne muscular dystrophy (DMD) were evaluated.
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