Update on Clinical Trial Endpoints in Gene Therapy Trials for Inherited Retinal Diseases.

Autor: Igoe, Jane M.¹ (AUTHOR) jane.igoe@gmail.com, Lam, Byron L.¹ (AUTHOR) blam@med.miami.edu, Gregori, Ninel Z.^1,2 (AUTHOR) ngregori@med.miami.edu

Publikováno v: Journal of Clinical Medicine. Sep2024, Vol. 13 Issue 18, p5512. 34p.

Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2/3 Study

Autor: MacLaren, Robert, Aleman, Tomas, Birch, David, Jalil, Assad, Lotery, Andrew, Lam, Byron, Pennesi, Mark, Kay, Christine N., Yusuf, Imram H., Cehajic Kapetanovic, Jasmina, Jolly, Jasleen K., Josan, Amandeep S., Taylor, Laura J., Xue, Kanmin, Nanda, Anika, Buckley, Thomas, Salvetti, Anna Paola, Thulasidharan, Suresh, Kurc, Miguel, Khandhadia, Samir, Dias, Karla Orsine Murta, Baltmr, Abeir, Mandal, Nakul, Tsokolas, Georgios, Ivanova, Tsveta, EL-Faouri, Muhannd, Shaarawy, Sherif, Black, Graeme, Davis, Janet L., Gregori, Ninel Z., Mendoza-Santiesteban, Carlos E., Lauer, Andreas K., Yang, Paul, Bailey, Steven, Spencer, Rand, Fish, Gary E., Wang, Robert, Chong, Deborah, Abbey, Ashkan, Anand, Rajiv, MaGuire, Albert A., Roseman, Robert L., Hazariwala, Kaushik M., Parrott, Brandon, Lam, Byron L., Pennesi, Mark E., Panda, Sushil, Gow, James A., Zhao, Guolin, MacLaren, Robert E.

Publikováno v: In Ophthalmology September 2024 131(9):1083-1093

XOLARIS: A 24-Month, Prospective, Natural History Study of 201 Participants with Retinitis Pigmentosa GTPase Regulator-Associated X-Linked Retinitis Pigmentosa

Autor: Gregory-Evans, Kevin, Koenekoop, Robert, Sankila, Eeva-Marja K., Bygglin, Henrik, Seitsonen, Sanna, Riikonen, Antti, Meunier, Isabelle, Fischer, M. Dominik, Ochakovski, Alex, Stingl, Katarina, Vaheb, Yousof, Richter, Paul, Wozar, Fabian, Reichel, Felix, Gassel, Caroline, Wolfram, Lasse, Fischer, Nora, Peters, Tobias, Wilhelm, Barbara, Seitz, Immanuel, Holz, Frank, Reinking, Katharina, Clemens, Amelie, Völker, Desiree, Herrmann, Philipp, Birtel, Johannes, Schipper, Pascal, Weber, Constance, Bulirsch, Louisa, Hoyng, Carel, Klaver, Caroline, Phan, T.M.L., Van Huet, Ramon, Boon, Camiel, Nguyen, X.T., Talib, M., Trzcionkowska, Kasia, Tussenbroek, Thomas, MacLaren, Robert E., Taylor, Laura J., Cehajic-Kapetanovic, Jasmina, Josan, Amandeep S., Yusuf, Imran H., Jasani, Kirti, Menghini, Moreno, Nanda, Anika, Rehman, Salwah, Jolly, Jasleen K., Buckley, Thomas M.W., Lotery, Andrew, Thulsidharan, Suresh, Khandhadia, Samir, Tsokolas, Georgios, Black, Graeme, Megaw, Roly, Bishop, Paul, Mukherjee, Rajarshi, Mohla, Aditi, McKibbin, Martin, Mukherjee, Raj, Lam, Byron L., Mendoza-Santiesteban, Carlos, Horowitz, Jason, Tsang, Stephen, Pennesi, Mark E., Yang, Paul, Lauer, Andreas K., Weleber, Richard G., Birch, David, Coors, Lori, Spencer, Rand, Csaky, Karl, Anand, Rajiv, Wang, Yi-Zhong, Gorin, Michael, Stepien, Kimberly, Duncan, Jacque L., Stewart, Jay, Moore, Anthony, Stout, J. Timothy, Weng, Christina, Leung, Ella, Schlle, Tahira, Bakall, Benjamin, Klein, Kendra, Bernstein, Paul, Hartnett, Mary Elizabeth, Mathias, Marc, Siringo, Frank, Pecen, Paula, Aleman, Tomas, McGuire, Albert, Nagiel, Aaron, Larsen, Michael, Ferraz Sallum, Juliana Maria, Ribeiro, Lucas, de Azevedo Amaral, Rebeca, Gow, James A., Li, Jiang, Tsang, So-Fai

Publikováno v: In Ophthalmology Science January-February 2025 5(1)

Leber Hereditary Optic Neuropathy Gene Therapy: Longitudinal Relationships Among Visual Function and Anatomical Measures

Autor: Lam, Byron L., Feuer, William J., Porciatti, Vittorio, Davis, Janet L., Zheng, D. Diane, Vanner, Elizabeth A., Savatovsky, Eleonore J., Alba, Diego E., Guy, John

Publikováno v: In American Journal of Ophthalmology January 2024 257:113-128

Phase I NT-501 Ciliary Neurotrophic Factor Implant Trial for Primary Open-Angle Glaucoma: Safety, Neuroprotection, and Neuroenhancement

Autor: Goldberg, Jeffrey L., Beykin, Gala, Satterfield, Kellie R., Nuñez, Mariana, Lam, Byron L., Albini, Thomas A.

Publikováno v: In Ophthalmology Science September 2023 3(3)