1Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104 USA
2Neurogene Inc., New York, NY USA
3Department of Medicine, Orphan Disease Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104 USA
4Translational Medicine & Human Genetics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104 USA
5Departments of Neurology and Pediatrics, Children’s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104 USA
6Division of Neurology, Children’s Hospital of Philadelphia, Philadelphia, PA 19104 USA
7Amicus Therapeutics, Philadelphia, PA 19104 USA
© The Author(s) 2023
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