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EMA Orphan Designation reflects high unmet need in SORD Deficiency and benefit of govorestat treatment in reducing toxic sorbitol levelsNew data published in Journal of Clinical Investigation further. | May 25, 2023 ....
AN2 Therapeutics, Inc. , a clinical-stage biopharmaceutical company focused on developing treatments for rare, chronic, and serious infectious diseases with high unmet needs, today announced that. | May 25, 2023 ....
RMAT recognizes that the preliminary clinical evidence from RGX-121, a potential one-time AAV Therapeutic, indicates the potential to address unmet medical needs for MPS II RMAT designation is for. | May 23, 2023 ....