Zi-Fan Yu

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Stephen R. Russell, Jean Bennett, Jennifer Wellman et al. · The Lancet · 2017 · 1.9K citations

Medicine, Ophthalmology, Vision Science +14

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

Paul Harmatz, Roberto Giugliani, Ida Vanessa Döederlein Schwartz et al. · Molecular Genetics and Metabolism · 2008 · 216 citations

Life Sciences, Biochemistry, Clinical Biochemistry +8

Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure

Johanna H. van der Lee, J. Morton, Heather Adams et al. · Molecular Genetics and Metabolism · 2017 · 47 citations · Full text

Medicine, Neurology, Pediatrics +17