Clinical TrialsEnrollment is now complete in the pivotal Phase 2 trial of RP-A501 in Danon disease, showcasing strong demand and setting the stage for a potential pivotal readout.
Manufacturing And InvestmentSignificant investments in manufacturing support all programs, demonstrating the company's commitment to advancing its pipeline.
Regulatory ApprovalThe company carefully selected indications with definable endpoints, ensuring that data from clinical trials could clearly demonstrate benefits to health authorities, paving the way for regulatory approval.