First in-human, intracisternal dosing of RGX-111, an investigational AAV gene therapy, for a 21-month-old child with mucopolysaccharidosis type I (MPS I): 3.5 year follow-upRaymond Wang, Laura Rosa Pisani, Mery Taylor et al.|Molecular Genetics and Metabolism|2024Cited by 1
First-in-human intracisternal dosing of RGX-111 in severe MPS I is well tolerated and generates sustained neurodevelopment without HSCTRaymond Wang, Laura Rosa Pisani, Nina Movsesyan et al.|Molecular Therapy|2026Cited by 0