Data RobustnessEarly efficacy signals were generated from very small patient groups, making the estimated treatment effect fragile and raising the risk that results may not hold in a larger, properly powered trial.
Funding RiskHigh annual cash burn and a limited cash runway increase the likelihood of financing pressure that could delay development milestones or lead to shareholder dilution before key approvals are achieved.
Reimbursement And Commercial RiskKey opinion leaders indicate that myotonia is not the primary burden for many patients, creating a risk that payers may not provide broad coverage if approval relies mainly on myotonia measures.