Clinical DataKRESLADI demonstrated a robust 100% survival rate beyond 1 year and beyond age 2 in all patients treated, which is significant for LAD-I patients.
Financial PerformanceRocket’s stock has been impacted by the broader market’s weakness rather than any change in fundamentals, suggesting its current valuation is not reflective of its potential.
Regulatory ApprovalThe approval of Kresladi for severe leukocyte adhesion deficiency-I is anticipated, which could significantly boost the company's portfolio.