Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation.
Valerio Carelli,
Nancy J Newman,
Patrick Yu-Wai-Man,
Valerie Biousse,
Mark L Moster,
Prem S Subramanian,
Catherine Vignal-Clermont,
An-Guor Wang,
Sean P Donahue,
Bart P Leroy,
Robert C Sergott,
Thomas Klopstock,
Alfredo A Sadun,
Gema Rebolleda Fernández,
Bart K Chwalisz,
Rudrani Banik,
Jean François Girmens,
Chiara La Morgia,
Adam A DeBusk,
Neringa Jurkute,
Claudia Priglinger,
Rustum Karanjia,
Constant Josse,
Julie Salzmann,
François Montestruc,
Michel Roux,
Magali Taiel,
José-Alain Sahel,
the LHON Study Group
OAI: oai:www.repository.cam.ac.uk:1810/345292 • DOI: 10.17863/CAM.92715