Clinical TrialsBB-301 low-dose regimen continues to demonstrate promise, with increasing durability of response at latest update.
Regulatory ApprovalAnalysts see a straightforward path to an approvable profile for BB-301 in oculopharyngeal muscular dystrophy, where current treatment options are limited and invasive.
Safety ProfileNo severe adverse events have been reported in any study subjects, suggesting a strong safety record for BB-301.