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REGENXBIO Announces FDA Review Extension of BLA for RGX-121 to Treat Patients with MPS II

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Summary

RGX-121 would be the first and only potential one-time commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome, if approved Commercial launch plans remain on track REGENXBIO plans to present updated pivotal data during the ICIEM meeting...

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Article published on August 18, 2025 by prnewswire