Muscular Dystrophy

i. Epidemiology of the disease. … a. What is the disease/condition in general terms? b. How many people are affected? What is the prevalence? c. Who gets this disease or has this condition? i. Differences among age, race, gender, economic status… d. Quality of life, Morbidity and Mortality data. e. What are the costs associated with the condition nationally? For the individual? t Local, National, and/or International statistics to demonstrate prevalence and scope of the disease.

H. Pathophysiology of the disease. What does this disease/condition look like in terms of severity of its impact on the individual? a. What is the disease/condition from a scientific perspective? i. Detailed relevant physiology, cellular biology, biochemistry and molecular biology (genetics) which illuminates the condition. b. Signs/Symptoms? How is it diagnosed? What are the common treatments (pharma, therapy, procedures)? c. What are the proposed mechanisms for why the most common treatments work? d. What are the risk factors for the disease/condition?

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