Question

Duchenne muscular dystrophy is an X-linked recessive disorder that affects approximately 1 in 3,600 boys. A new drug has recently been developed to slow the progression of disease, which has little carry-over effects if the patient stops taking the drug. Because the rate of improvement varies substantially between patients and since this is a rare condition that only affects a small population, researchers seek a study design that reduces variability between patients and utilizes a limited sample size. The primary research question of interest is whether this new drug is more efficacious than the standard of care. Which study design best describes this trial?

Answer 1

Answer:

Duchenne muscular dystrophy (DMD) is a disease linked to the X-chromosome which affects 1 in 3,600-6,000 newborn males. It is manifested by the absence of the dystrophin protein in muscle fibres, which causes progressive damage leading to death in the third decade of life. The only medication so far shown to be effective in delaying the progression of this illness

Explanation:

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