Hunter syndrome

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Biotech

Regenxbio gene therapy refiling plan derailed by FDA hold

The FDA put a hold on RGX-121 over spine MRI findings, derailing the biotech’s plans to refile for approval of the once-rejected gene therapy.

Nick Paul Taylor Aug 24, 2026 7:51am

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Fierce Pharma

FDA approves Denali's Hunter syndrome drug

Mar 25, 2026 2:50pm

MPS Families Carry Coffin to FDA

Rare disease advocates stage 'funeral' at FDA, make 4 demands

Mar 18, 2026 5:25pm

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FDA details Regenxbio rare disease rejection

Mar 3, 2026 12:56pm

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FDA rejects Regenxbio's Hunter syndrome gene therapy

Feb 9, 2026 4:05pm

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Royalty's $275M financing deal for Denali drug awaiting FDA nod

Dec 4, 2025 10:20am