DMD CLINICAL THERAPIES II

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Duchenne muscular dystrophy (DMD) patients experience progressive degeneration of skeletal muscles including those involved in respiration, and respiratory decline is linked to mortality. Onset of respiratory decline precedes increasing levels of clinical management as FVC%p falls below established thresholds. The goal of treatment is to delay time to these thresholds. While motor function in eteplirsen-treated patients in study 201/202 diverges from natural history after 1 year of treatment, it is unclear if respiratory function follows a similar pattern.

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