FDA pauses Regenxbio Hunter syndrome gene therapy after spine masses detected in five patients
TL;DR Summary
The FDA has paused Regenxbio's gene therapy trial for Hunter syndrome (MPS II) after five trial participants were found to have small, asymptomatic masses on their spines, highlighting ongoing safety concerns with AAV-based gene therapies and echoing a prior brain-tumor case linked to a similar approach.
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