Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy
Catherine Vignal, Patrick Yu‐Wai‐Man, Nancy J. Newman, Valério Carelli, Mark L. Moster, Valérie Biousse, Prem S. Subramanian, An-Guor Wang, Sean P. Donahue, Bart P. Leroy, Alfredo A. Sadun, Thomas Klopstock, Robert C. Sergott, Gema Rebolleda Fernández, Bart K. Chwalisz, Rudrani Banik, Magali Taiel, Michel J. Roux, José‐Alain Sahel
Topics & Concepts
MedicineTolerabilityDiscontinuationIntraocular pressureAdverse effectOptic neuropathyOphthalmologyOptic nerveInternal medicineSurgeryRetinal Development and DisordersVirus-based gene therapy researchEndoplasmic Reticulum Stress and Disease