Orchard Therapeutics Announces Presentation of Data Comprising the Clinical Package for the OTL-200 BLA in MLD at the SSIEM Annual Symposium 2023 [Yahoo! Finance]
Orchard Therapeutics plc - American Depositary Shares (ORTX)
Last orchard therapeutics plc - american depositary shares earnings: 2/27 07:00 am
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Source: Yahoo! Finance
Administration of one-time gene therapy resulted in statistically significant improvement in severe motor impairment-free survival with up to 12 years of follow-up (median 6.76 years) Composite primary endpoint used in analysis was developed through discussions with the U.S. Food and Drug Administration Extended follow-up continues to demonstrate sustained preservation of cognitive function and motor development in most patients BOSTON and LONDON, Aug. 31, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced long-term results from an updated integrated analysis of 39 patients with metachromatic leukodystrophy (MLD) treated with investigational OTL-200 in the clinical development program. The data were presented yesterday at the ongoing Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem. “MLD is a devastating and ultimately fatal disease for which there are no alternative treatment o
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