News and commentary from the world of neurology and neuroscience
— News and commentary from the world of neurology and neuroscience
by Judy George, Deputy Managing Editor, MedPage Today
June 23, 2026
• 2 min read
Gene therapy developer uniQure said it will submit a marketing application for accelerated approval for investigational AMT-130 for Huntington's disease after the FDA reversed its position and agreed that a 3-year analysis of a phase I/II study showing benefit was acceptable.
FDA reverses course on Regenxbio’s childhood gene therapy after rejection
Regenxbio to resubmit gene therapy as FDA backtracks on another drug rejection
Regenxbio Gains Fresh Chance As FDA Reconsiders Gene Therapy - Regenxbio (NASDAQ:RGNX)
Wagering on FDA changes, Regenxbio will submit Duchenne gene therapy for approval
Regenxbio to resubmit Hunter syndrome gene therapy as FDA rare disease about-face continues
REGENXBIO Announces Alignment with FDA on Path Forward for NAVSUNLI™ BLA Resubmission for Accelerated Approval; First Potential Gene Therapy for MPS II
The FDA will reconsider approving Regenxbio's experimental gene therapy for a deadly and rare childhood brain disorder it…
News and commentary from the world of neurology and neuroscience
The FDA has reversed its opposition to a closely watched experimental treatment for Huntington’s disease, clearing a path for its…
Vinay Prasad's acting replacement, remembering genomics pioneer J. Craig Venter, and more biotech news from The Readout
News and commentary from the world of neurology and neuroscience
The decision marks another regulatory U-turn following the exits of Marty Makary and Vinay Prasad, suggesting to some analysts…
