Clinical Trial SuccessEWTX's GRAND CANYON study appears well-suited to achieve a significant difference in NSAA, with >98% power to observe a significant difference between treatment and placebo.
Drug DevelopmentSevasemten aims to become the first approved drug for Becker muscular dystrophy and an adjuvant for Duchenne muscular dystrophy standard of care.
Product DifferentiationSevasemten offers a mutation agnostic approach for dystrophin- or dystroglycan complex-related disorders, with limited potential for serious off-target side effects.
Regulatory ProgressThe FDA has provided a clear path to registration for sevasemten in Becker muscular dystrophy.