Duchenne muscular dystrophy (DMD) causes progressive muscle degeneration. An unmet need exists for a standardized, validated observer-rated instrument to augment efficacy and safety assessments in clinical trials of DMD therapies and further elucidate potential risks and benefits of treatment. Recently, regulatory authorities such as the European Medicines Agency and Food and Drug Administration clarified the possible add-on value of collecting such data in drug development. Two observer-reported outcome measures will be evaluated in the double-blind, randomized, placebo-controlled phase 3 ESSENCE trial (NCT02500381) of 2 investigational exon-skipping therapeutics in ambulant patients with DMD gene variants amenable to skipping exon 45 or 53.

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