Dossier
mucopolysaccharidosis type II
Coverage of mucopolysaccharidosis type II in the Nexus archive.
- STAT+: FDA reverses course on Regenxbio’s childhood gene therapy after rejection
The FDA will reconsider approving Regenxbio’s gene therapy for a rare childhood brain disorder, mucopolysaccharidosis type II (MPS II), after initially rejecting it four months ago. This follows recent FDA reversals, including allowing UniQure to resubmit a gene therapy application for Huntington’s disease previously rejected by former commissioner Marty Makary.