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US FDA accepts and grants priority review status to Regenxbio’s BLA for clemidsogene lanparvovec to treat Mucopolysaccharidosis II: Rockville, Maryland Thursday, May 15, 2025, 1 ...
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DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy DesignationDenali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).
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