Clinical TrialsFORTITUDE trial data showed directional improvement in key clinical endpoints like RWS, total composite score, shoulder abductor, and ankle dorsiflexion, when compared to the placebo arm.
Financial StabilityStrong cash runway supports a rich and steady pipeline development.
Regulatory ApprovalRNA has built flexibility into their approach to accelerated approval in FSHD which could help them gain alignment with the FDA.