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This video explores the most common muscular dystrophy in children called Duchenne muscular dystrophy (DMD), a genetic disorder. DMD is explained through signs and symptoms, pathogenesis, diagnosis and disease progression. Studies were used to explain the benefits of exercise as well as treatment with Deflazacort.
This video was made by Demystifying Medicine students Lamisha Khandaker, Kunal Mehta, Michelle Rong, and Dhruvika Joshi.
Copyright McMaster University 2018
If you'd like to learn more about DMD and how you can help, please visit:
www.mda.org/disease/duchenne-...
Here are the 2 studies mentioned in the video:
Study on Delflazacort:
Griggs, R. C., Miller, J. P., Greenberg, C. R., Fehlings, D. L., Pestronk, A., Mendell, J. R., . . . Meyer, J. M. (2016, November 15). Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Retrieved January 18, 2018, from n.neurology.org/content/87/20/...
Study on Exercise:
Jansen, M., Alfen, N. V., Geurts, A. C., & Groot, I. J. (2013, July 24). Assisted Bicycle Training Delays Functional Deterioration in Boys With Duchenne Muscular Dystrophy: The Randomized Controlled Trial "No Use Is Disuse". Retrieved January 18, 2018, from www.researchgate.net/publicat...
References:
Le Guiner, C., Servais, L., Montus, M., Larcher, T., Fraysse, B., Moullec, S., ... & Koo, T. (2017, July 25). Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. Retrieved January 18, 2018, from www.nature.com/articles/ncomm...
McDonald, C. M., Henricson, E. K., Abresch, R. T., Florence, J., Eagle, M., Gappmaier, E., . . . Peltz, S. W. (2013, July 17). 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. Retrieved January 18, 2018, from onlinelibrary.wiley.com/doi/10...
Ropars, J., Lempereur, M., Vuillerot, C., Tiffreau, V., Peudenier, S., Cuisset, J., . . . Brochard, S. (2016, September 13). Muscle Activation during Gait in Children with Duchenne Muscular Dystrophy. Retrieved January 18, 2018, from journals.plos.org/plosone/arti...
Ruiten, H. J., Straub, V., Bushby, K., & Guglieri, M. (2014, September 3). Improving recognition of Duchenne muscular dystrophy: a retrospective case note review. Retrieved January 18, 2018, from adc.bmj.com/content/early/2014...
Sienkiewicz, D., Kulak, W., Okurowska-Zawada, B., Paszko-Patej, G., & Kawnik, K. (2015, July). Duchenne muscular dystrophy: current cell therapies. Retrieved January 18, 2018, from www.ncbi.nlm.nih.gov/pmc/arti...
Sussman, M. (2002). Duchenne Muscular Dystrophy. Journal of the American Academy of Orthopaedic Surgeons, 10(2), 138-151.
Vohra, R. S., Lott, D., Mathur, S., Senesac, C., Deol, J., Germain, S., . . . Vandenborne, K. (2015, June 23). Magnetic Resonance Assessment of Hypertrophic and Pseudo-Hypertrophic Changes in Lower Leg Muscles of Boys with Duchenne Muscular Dystrophy and Their Relationship to Functional Measurements. Retrieved January 18, 2018, from journals.plos.org/plosone/arti...