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- FDA pauses Regenxbio gene therapy trial for Hunter syndrome after spinal masses foundThe Food and Drug Administration has placed another hold on Regenxbio's gene-therapy trial for mucopolysaccharidosis type II, also known as Hunter syndrome, after new safety concerns emerged. Five…
- 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…