Autosomal Dominant Retinitis Pigmentosa Due to Class B Rhodopsin Mutations: An Objective Outcome for Future Treatment Trials

por Sumaroka, Alexander, Cideciyan, Artur V., Charng, Jason, Wu, Vivian, Powers, Christian A., Iyer, Bhavya S., Lisi, Brianna, Swider, Malgorzata, Jacobson, Samuel G.
Publicado 2019

Short-Wavelength Sensitive Cone (S-cone) Testing as an Outcome Measure for NR2E3 Clinical Treatment Trials

por Roman, Alejandro J., Powers, Christian A., Semenov, Evelyn P., Sheplock, Rebecca, Aksianiuk, Valeryia, Russell, Robert C., Sumaroka, Alexander, Garafalo, Alexandra V., Cideciyan, Artur V., Jacobson, Samuel G.
Publicado 2019

Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial

por Russell, Stephen R., Drack, Arlene V., Cideciyan, Artur V., Jacobson, Samuel G., Leroy, Bart P., Van Cauwenbergh, Caroline, Ho, Allen C., Dumitrescu, Alina V., Han, Ian C., Martin, Mitchell, Pfeifer, Wanda L., Sohn, Elliott H., Walshire, Jean, Garafalo, Alexandra V., Krishnan, Arun K., Powers, Christian A., Sumaroka, Alexander, Roman, Alejandro J., Vanhonsebrouck, Eva, Jones, Eltanara, Nerinckx, Fanny, De Zaeytijd, Julie, Collin, Rob W. J., Hoyng, Carel, Adamson, Peter, Cheetham, Michael E., Schwartz, Michael R., den Hollander, Wilhelmina, Asmus, Friedrich, Platenburg, Gerard, Rodman, David, Girach, Aniz
Publicado 2022