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Orchard Therapeutics Announced Long-term Results From An Updated Integrated Analysis Of 39 Patients With Metachromatic Leukodystrophy Treated With Investigational OTL-200 In The Clinical Development Program

Administration of one-time gene therapy resulted in statistically significant improvement in severe motor impairment-free survival with up to 11 years of follow-up (median 6.15 years) Composite primary endpoint used

ORTX