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Mps Ii

All articles tagged with #mps ii

health2 hours ago•2 min saved

FDA pauses Regenxbio Hunter syndrome gene therapy after spine masses detected in five patients

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.

via STAT|

#fda #gene-therapy #health

health2 months ago•2 min saved

FDA reopens review of Regenxbio's Hunter syndrome gene therapy

The FDA is reconsidering Regenxbio’s experimental gene therapy for mucopolysaccharidosis type II (Hunter syndrome) after previously rejecting it, part of a series of recent agency reversals amid leadership changes; the case follows another reversal where UniQure moved to submit a Huntington’s disease gene therapy previously spurned by the agency.

via statnews.com|

#fda #gene-therapy #health