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AJ201 demonstrated positive Phase 1/2a results in SBMA patients, showing clinically meaningful improvements in physical and muscle function, with a 17.6-meter gain in 6-Minute Walk Test and 0.8-point increase in SBMA Functional Rating Scale compared to placebo.[10] Notably, nuclear mutant androgen receptor levels were reduced by more than 50% in 53% of AJ201-treated patients versus 17% of placebo, with RNA sequencing confirming Nrf2 pathway activation.[10]
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