Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

por Day, John W., Finkel, Richard S., Mercuri, Eugenio, Swoboda, Kathryn J., Menier, Melissa, van Olden, Rudolf, Tauscher-Wisniewski, Sitra, Mendell, Jerry R.
Publicado 2021

Single‐ and multiple‐dose safety, tolerability, pharmacokinetic, and pharmacodynamic profiles of ASP0367, or bocidelpar sulfate, a novel modulator of peroxisome proliferator‐activa...

por Ito, Mototsugu, Tauscher‐Wisniewski, Sitra, Smulders, Ronald A., Wojtkowski, Tomasz, Yamada, Akihiro, Koibuchi, Akira, Uz, Tolga, Marek, Gerard J., Goldwater, Ronald D.
Publicado 2021

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

por Day, John W., Mendell, Jerry R., Mercuri, Eugenio, Finkel, Richard S., Strauss, Kevin A., Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P., Chand, Deepa H.
Publicado 2021

Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

por Day, John W., Mendell, Jerry R., Mercuri, Eugenio, Finkel, Richard S., Strauss, Kevin A., Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P., Chand, Deepa H.
Publicado 2021

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

por Finkel, Richard S., Darras, Basil T., Mendell, Jerry R., Day, John W., Kuntz, Nancy L., Connolly, Anne M., Zaidman, Craig M., Crawford, Thomas O., Butterfield, Russell J., Shieh, Perry B., Tennekoon, Gihan, Brandsema, John F., Iannaccone, Susan T., Shoffner, John, Kavanagh, Sarah, Macek, Thomas A., Tauscher-Wisniewski, Sitra
Publicado 2023

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

por Davidson, Beverly L., Gao, Guangping, Berry-Kravis, Elizabeth, Bradbury, Allison M., Bönnemann, Carsten, Buxbaum, Joseph D., Corcoran, Gavin R., Gray, Steven J., Gray-Edwards, Heather, Kleiman, Robin J., Shaywitz, Adam J., Wang, Dan, Zoghbi, Huda Y., Flotte, Terence R., Tauscher-Wisniewski, Sitra, Tifft, Cynthia J., Sahin, Mustafa
Publicado 2022

Determining Pharmacological Selectivity of the Kappa Opioid Receptor Antagonist LY2456302 Using Pupillometry as a Translational Biomarker in Rat and Human

por Rorick-Kehn, Linda M., Witcher, Jennifer W., Lowe, Stephen L., Gonzales, Celedon R., Weller, Mary Ann, Bell, Robert L., Hart, John C., Need, Anne B., McKinzie, Jamie H., Statnick, Michael A., Suico, Jeffrey G., McKinzie, David L., Tauscher-Wisniewski, Sitra, Mitch, Charles H., Stoltz, Randall R., Wong, Conrad J.
Publicado 2015

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

por Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.
Publicado 2022

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

por Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Wigderson, Melissa, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.
Publicado 2022