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The FDA Places Clinical Hold On Regenxbio's RGX-121 Following Asymptomatic Spine MRI Findings In Five Participants In CAMPSIITE Study In Mucopolysaccharidosis Type II Patients

Aug 24, 2026 · 07:11 AM ET· updated 1h ago

REGENXBIO Inc. (NASDAQ:RGNX) today provided an update on its investigational gene therapy, RGX-121 (clemidsogene lanparvovec), for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter Syndrome.

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Why It Matters

The FDA paused testing of a new medicine that was supposed to help kids with a rare disease because doctors saw unexpected spine changes on scans, even though the kids felt fine. When a medicine gets paused like this, it's a sign the company needs to figure out what happened and prove it's safe before continuing.

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Source: Benzinga · Read the original report at the publisher. Headline and figures shown for context; data may be delayed.

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