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- FDA places hold on REGENXBIO's Hunter syndrome gene therapy after spinal nodules found, company pauses resubmissionThe FDA placed a clinical hold on RGX-121, REGENXBIO's gene therapy for Hunter syndrome, after MRI scans showed small nodules or cystic masses in the spines of five trial participants. The finding…
- FDA’s new leadership aims to restore reliability, but each application remains judged on its dataThe FDA’s current leadership is working to rebuild the agency’s reputation for reliability, integrity and transparency after a turbulent period under former commissioner Marty Makary. Recent actio…