Single‐ and multiple‐dose safety, tolerability, pharmacokinetic, and pharmacodynamic profiles of ASP0367, or bocidelpar sulfate, a novel modulator of peroxisome proliferator‐activa... von Ito, Mototsugu, Tauscher‐Wisniewski, Sitra, Smulders, Ronald A., Wojtkowski, Tomasz, Yamada, Akihiro, Koibuchi, Akira, Uz, Tolga, Marek, Gerard J., Goldwater, Ronald D.

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Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy von Chand, Deepa, Mohr, Franziska, McMillan, Hugh, Tukov, Francis Fonyuy, Montgomery, Kyle, Kleyn, Aaron, Sun, Rui, Tauscher-Wisniewski, Sitra, Kaufmann, Petra, Kullak-Ublick, Gerd

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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial von Mercuri, Eugenio, Muntoni, Francesco, Baranello, Giovanni, Masson, Riccardo, Boespflug-Tanguy, Odile, Bruno, Claudio, Corti, Stefania, Daron, Aurore, Deconinck, Nicolas, Servais, Laurent, Straub, Volker, Ouyang, Haojun, Chand, Deepa, Tauscher-Wisniewski, Sitra, Mendonca, Nuno, Lavrov, Arseniy, Seferian, A., De Lucia, S., Tachibana, S., Jollet, A., Mouffak, S., Pedemonte, M., Brolatti, N., Morando, S., Vanlander, A., De Vos, E., Tahon, V., Govoni, A., Magri, F., Comi, G., Foa, M., Parente, V., Buscemi, L., Dal Farra, F., Schneider, O., Jonas, A., Defeldre, A.C., Pagliano, E., Zanin, R., Arnoldi, M.T., Schembri, V., Del Sole, M., Mandelli, A., Pera, M.C., Antonaci, L., Coratti, G., de Sanctis, R., Pane, M., Scoto, M., Groves, K., Edel, L., Abel, F., Van Ruiten, H., Lofra, R.M., Thompson, E.

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Identification and validation of biomarkers for autism spectrum disorders von Loth, Eva, Spooren, Will, Ham, Lindsay M., Isaac, Maria B., Auriche-Benichou, Caroline, Banaschewski, Tobias, Baron-Cohen, Simon, Broich, Karl, Bölte, Sven, Bourgeron, Thomas, Charman, Tony, Collier, David, de Andres-Trelles, Fernando, Durston, Sarah, Ecker, Christine, Elferink, Andre, Haberkamp, Marion, Hemmings, Robert, Johnson, Mark H., Jones, Emily J. H., Khwaja, Omar S., Lenton, Sabine, Mason, Luke, Mantua, Valentina, Meyer-Lindenberg, Andreas, Lombardo, Michael V., O'Dwyer, Laurence, Okamoto, Koichi, Pandina, Gahan J., Pani, Luca, Persico, Antonio M., Simonoff, Emily, Tauscher-Wisniewski, Sitra, Llinares-Garcia, Jordi, Vamvakas, Spiros, Williams, Steve, Buitelaar, Jan K., Murphy, Declan G. M.

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Evaluation of single and multiple doses of a novel mGlu2 agonist, a potential antipsychotic therapy, in healthy subjects von McColm, Juliet, Brittain, Claire, Suriyapperuma, Subha, Swanson, Steven, Tauscher‐Wisniewski, Sitra, Foster, Joanne, Soon, Danny, Jackson, Kimberley

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Safety, tolerability, and pharmacokinetic evaluation of single- and multiple-ascending doses of a novel kappa opioid receptor antagonist LY2456302 and drug interaction with ethanol... von Lowe, Stephen L., Wong, Conrad J., Witcher, Jennifer, Gonzales, Celedon R., Dickinson, Gemma L., Bell, Robert L., Rorick-Kehn, Linda, Weller, MaryAnn, Stoltz, Randall R., Royalty, Jane, Tauscher-Wisniewski, Sitra

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Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec von Day, John W., Finkel, Richard S., Mercuri, Eugenio, Swoboda, Kathryn J., Menier, Melissa, van Olden, Rudolf, Tauscher-Wisniewski, Sitra, Mendell, Jerry R.

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Meta-Analysis of Suicide-Related Behavior Events in Patients Treated With Atomoxetine von BANGS, MARK E., M.D, TAUSCHER-WISNIEWSKI, SITRA, M.D, POLZER, JOHN, D.V.M, ZHANG, SHUYU, M.S, ACHARYA, NAYAN, M.B.B.S, DESAIAH, DURISALA, Ph.D, TRZEPACZ, PAULA T., M.D, ALLEN, ALBERT J., M.D., Ph.D

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Cortisol response to individualised graded insulin infusions: a reproducible biomarker for CNS compounds inhibiting HPA activation von Chen, Christopher L. H., Willis, Brian A., Mooney, Louise, Ong, Guan Koon, Lim, Chay Ngee, Lowe, Stephen L., Tauscher‐Wisniewski, Sitra, Cutler Jr, Gordon B., Wiss, Stephen D.

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Meta-Analysis of Aggression or Hostility Events in Randomized, Controlled Clinical Trials of Atomoxetine for ADHD von Polzer, John, Bangs, Mark E, Zhang, Shuyu, Dellva, Mary Anne, Tauscher-Wisniewski, Sitra, Acharya, Nayan, Watson, Susan B, Allen, Albert J, Wilens, Timothy E

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

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

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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre,... von Day, John W, Finkel, Richard S, Chiriboga, Claudia A, Connolly, Anne M, Crawford, Thomas O, Darras, Basil T, Iannaccone, Susan T, Kuntz, Nancy L, Peña, Loren D M, Shieh, Perry B, Smith, Edward C, Kwon, Jennifer M, Zaidman, Craig M, Schultz, Meredith, Feltner, Douglas E, Tauscher-Wisniewski, Sitra, Ouyang, Haojun, Chand, Deepa H, Sproule, Douglas M, Macek, Thomas A, Mendell, Jerry R

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Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy von 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.

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Long-Term Follow-Up (LTFU) of Onasemnogene Abeparvovec Gene Therapy in Spinal Muscular Atrophy (SMA) (2301) von Mendell, Jerry, Finkel, Richard, Mercuri, Eugenio, Strauss, Kevin, Day, John, Kleyn, Aaron, Chand, Deepa, Tauscher-Wisniewski, Sitra, Meriggioli, Matthew

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CORRECTION von Day, John W, Mendel, Jerry R, Mercuri, Eugenio, Finkel, Richard S, Strauss, Kevin A, Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P, Chand, Deepa H

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Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy von Chand, Deepa, Mohr, Franziska, McMillan, Hugh, Tukov, Francis Fonyuy, Montgomery, Kyle, Kleyn, Aaron, Sun, Rui, Tauscher-Wisniewski, Sitra, Kaufmann, Petra, Kullak-Ublick, Gerd

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Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders von 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

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Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 3 Copies of SMN2 (4163) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, Macek, Thomas

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Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 2 Copies of SMN2 (4190) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, Macek, Tom

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