Search Results - "Wandel, Christoph"

Basmisanil, a highly selective GABAA-α5 negative allosteric modulator: preclinical pharmacology and demonstration of functional target engagement in man by Hipp, Joerg F., Knoflach, Frederic, Comley, Robert, Ballard, Theresa M., Honer, Michael, Trube, Gerhard, Gasser, Rodolfo, Prinssen, Eric, Wallace, Tanya L., Rothfuss, Andreas, Knust, Henner, Lennon-Chrimes, Sian, Derks, Michael, Bentley, Darren, Squassante, Lisa, Nave, Stephane, Nöldeke, Jana, Wandel, Christoph, Thomas, Andrew W., Hernandez, Maria-Clemencia

“…GABA A -α5 subunit-containing receptors have been shown to play a key modulatory role in cognition and represent a promising drug target for cognitive…”
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A Framework Proposal to Follow‐Up on Preclinical Convulsive Signals of a New Molecular Entity in First‐in‐Human Studies Using Electroencephalographic Monitoring by Abt, Markus, Dinklo, Theo, Rothfuss, Andreas, Husar, Elisabeth, Dannecker, Robert, Kallivroussis, Katja, Peck, Richard, Doessegger, Lucette, Wandel, Christoph

“…Traditionally, in dose‐escalating first‐in‐human (FiH) studies, a dose cap with a 10‐fold safety margin to the no observed effect level in animals is…”
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A randomized, double-blind, placebo-controlled phase II trial to explore the effects of a GABAA-α5 NAM (basmisanil) on intellectual disability associated with Down syndrome by Goeldner, Celia, Kishnani, Priya S., Skotko, Brian G., Casero, Julian Lirio, Hipp, Joerg F., Derks, Michael, Hernandez, Maria-Clemencia, Khwaja, Omar, Lennon-Chrimes, Sian, Noeldeke, Jana, Pellicer, Sabine, Squassante, Lisa, Visootsak, Jeannie, Wandel, Christoph, Fontoura, Paulo, d’Ardhuy, Xavier Liogier, De La Torre Fornell, Rafael, Glue, Paul, Hoover-Fong, Julie, Uhlmann, Sonja, Malagón Valdez, Jorge, Marshall, Andrew, Martinón-Torres, Federico, Redondo-Collazo, Lorenzo, Rodriguez-Tenreiro, Carmen, Marquez Chin, Valeria, Michel Reynoso, Adriana G., Mitchell, Ed A., Slykerman, Rebecca F., Wouldes, Trecia, Loveday, Sarah, Moldenhauer, Fernando, Novell, Ramon, Ochoa, Cesar, Rafii, Michael S., Rebillat, Anne-Sophie, Sanlaville, Damien, Sarda, Pierre, Shankar, Rohit, Pulsifer, Margaret, Evans, Casey L., Silva, Alexandra M., McDonough, Mary Ellen, Stanley, Maria, McCary, Lindsay M., Vicari, Stefano, Wilcox, William, Zampino, Giuseppe, Zuddas, Alessandro

“…Abstract Background There are currently no pharmacological therapies to address the intellectual disability associated with Down syndrome…”
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O19: Integrated analyses of data from clinical trials of delandistrogene moxeparvovec gene therapy in DMD by O’Rourke, Erin, Proud, Crystal, Zaidman, Craig, Shieh, Perry B., McDonald, Craig, Day, John W., Mason, Stefanie, Guridi, Maitea, Han, Lixin, Yu, Lixi, Reid, Carol, Darton, Eddie, Wandel, Christoph, Richardson, James, Malhotra, Jyoti, Singh, Teji, Rodino-Klapac, Louise, Mendell, Jerry

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Pharmacological Inhibition of P-glycoprotein Transport Enhances the Distribution of HIV-1 Protease Inhibitors into Brain and Testes by Choo, E F, Leake, B, Wandel, C, Imamura, H, Wood, A J, Wilkinson, G R, Kim, R B

“…HIV protease inhibitors have proven remarkably effective in treating HIV-1 infection. However, some tissues such as the brain and testes (sanctuary sites) are…”
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Interrelationship between substrates and inhibitors of human CYP3A and P-glycoprotein by KIM, R. B, WANDEL, C, WOOD, A. J. J, WILKINSON, G. R, LEAKE, B, CVETKOVIC, M, FROMM, M. F, DEMPSEY, P. J, RODEN, M. M, BELAS, F, CHAUDHARY, A. K, RODEN, D. M

“…CYP3A and P-gp both function to reduce the intracellular concentration of drug substrates, one by metabolism and the other by transmembrane efflux. Moreover,…”
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Clinical trial considerations on male contraception and collection of pregnancy information from female partners by Banholzer, Maria Longauer, Buergin, Heinrich, Wandel, Christoph, Schmitt, Georg, Gocke, Elmar, Peck, Richard, Singer, Thomas, Reynolds, Theresa, Mannino, Marie, Deutsch, Jonathan, Doessegger, Lucette

“…There is little guidance regarding the risk of exposure of pregnant women/ women of childbearing potential to genotoxic or teratogenic compounds via vaginal…”
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P4‐185: First in‐human PET study of 3 novel tau radiopharmaceuticals: [11C]RO6924963, [11C]RO6931643, and [18F]RO6958948 by Wong, Dean F., Borroni, Edilio, Kuwabara, Hiroto, George, Noble, Rosenberg, Paul, Lyketsos, Constantine, Resnick, Susan M., Thambisetty, Madhav, Brasic, James, Gapasin, Lorena, Willis, William, Knust, Henner, Guerard, Melanie, Belli, Sara, Muri, Dieter, Carey, Tracie, Bedding, Alun, Wandel, Christoph, Hansrod, Tasnim, Honer, Michael, Moghekar, Abhay, Boess, Frank, Albert, Marilyn S., Shaya, Elias, Oh, Esther, Ostrowitzki, Susanne, Dannals, Robert F., Comley, Robert A.

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Morbidity and medication in a large population of individuals with Down syndrome compared to the general population by Alexander, Myriam, Petri, Hans, Ding, Yingjie, Wandel, Christoph, Khwaja, Omar, Foskett, Nadia

“…Aim The aim of this study was to describe the incidence of morbidities and the prevalence of medical prescriptions in a large Down syndrome population. Method…”
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Balovaptan vs Placebo for Social Communication in Childhood Autism Spectrum Disorder: A Randomized Clinical Trial by Hollander, Eric, Jacob, Suma, Jou, Roger, McNamara, Nora, Sikich, Linmarie, Tobe, Russell, Smith, Janice, Sanders, Kevin, Squassante, Lisa, Murtagh, Lorraine, Gleissl, Teresa, Wandel, Christoph, Veenstra-VanderWeele, Jeremy

“…There are no approved medications for the core symptoms of autism spectrum disorder (ASD), socialization and communication difficulties. To evaluate the…”
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Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy by Mendell, Jerry R., Proud, Crystal, Zaidman, Craig M., Mason, Stefanie, Darton, Eddie, Wang, Shufang, Wandel, Christoph, Murphy, Alexander P., Mercuri, Eugenio, Muntoni, Francesco, McDonald, Craig M.

“…Delandistrogene moxeparvovec is a gene transfer therapy approved in the United States, United Arab Emirates, and Qatar for the treatment of ambulatory patients…”
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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR) by Zaidman, Craig M., Proud, Crystal M., McDonald, Craig M., Lehman, Kelly J., Goedeker, Natalie L., Mason, Stefanie, Murphy, Alexander P., Guridi, Maitea, Wang, Shufang, Reid, Carol, Darton, Eddie, Wandel, Christoph, Lewis, Sarah, Malhotra, Jyoti, Griffin, Danielle A., Potter, Rachael A., Rodino‐Klapac, Louise R., Mendell, Jerry R.

“…Objective Delandistrogene moxeparvovec is approved in the USA for the treatment of ambulatory patients (4–5 years) with Duchenne muscular dystrophy. ENDEAVOR…”
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Integrated Analyses of Data from Clinical Trials of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD) (S48.006) by Proud, Crystal, Zaidman, Craig, Shieh, Perry, McDonald, Craig, Day, John, Mason, Stefanie, Guridi, Maitea, Han, Lixin, Yu, Lixi, Reid, Carol, Darton, Eddie, Wandel, Christoph, Richardson, James, Malhotra, Jyoti, Singh, Tejdip, Rodino-Klapac, Louise, Mendell, Jerry

“…Abstract only…”
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EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP- 9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics (P5-8.012) by Muntoni, Francesco, Mercuri, Eugenio, Schmidt, Ulrike Schara, Komaki, Hirofumi, Richardson, James, Singh, Tejdip, Guridi, Maitea, Mason, Stefanie, Murphy, Alex, Yu, Lixi, Reid, Carol, Darton, Eddie, Wandel, Christoph, Mendell, Jerry

“…Abstract only…”
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One-year Data from ENDEAVOR, a Phase 1b Trial of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD) (S48.003) by Zaidman, Craig, Proud, Crystal, McDonald, Craig, Mason, Stefanie, Guridi, Maitea, Wang, Shufang, Reid, Carol, Darton, Eddie, Wandel, Christoph, Lewis, Sarah, Malhotra, Jyoti, Griffin, Danielle, Potter, Rachael, Rodino-Klapac, Louise, Mendell, Jerry

“…Abstract only…”
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Efficacy and safety of balovaptan for socialisation and communication difficulties in autistic adults in North America and Europe: a phase 3, randomised, placebo-controlled trial by Jacob, Suma, Veenstra-VanderWeele, Jeremy, Murphy, Declan, McCracken, James, Smith, Janice, Sanders, Kevin, Meyenberg, Christoph, Wiese, Thomas, Deol-Bhullar, Gurpreet, Wandel, Christoph, Ashford, Elizabeth, Anagnostou, Evdokia

“…There are no approved pharmacological therapies to support treatment of the core communication and socialisation difficulties associated with autism spectrum…”
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6.14 PHASE 3 RANDOMIZED CONTROLLED TRIAL OF BALOVAPTAN IN ADULTS WITH AUTISM SPECTRUM DISORDER by Jacob, Suma, Veenstra-VanderWeele, Jeremy, Murphy, Declan, McCracken, James T., Smith, Janice, Sanders, Kevin, Meyenberg, Christoph, Wiese, Thomas, Deol-Bhullar, Gurpreet, Wandel, Christoph, Anagnostou, Evdokia

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A PHASE 2 RANDOMIZED CONTROLLED TRIAL OF BALOVAPTAN IN PEDIATRIC PARTICIPANTS WITH AUTISM SPECTRUM DISORDER by Hollander, Eric, Jacob, Suma, Jou, Roger J., McNamara, Nora, Sikich, Linmarie, Tobe, Russell, Smith, Janice, Sanders, Kevin, Squassante, Lisa, Murtagh, Lorraine, Gleissl, Teresa, Wandel, Christoph, Veenstra-VanderWeele, Jeremy

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PKPD and cardiac single cell modeling of a DDI study with a CYP3A4 substrate and itraconazole to quantify the effects on QT interval duration by Jaminion, Felix, Bentley, Darren, Wang, Ken, Wandel, Christoph, Derks, Michael, Diack, Cheikh

“…Plasma drug concentration and electrocardiogram (ECG) data from a drug–drug interaction (DDI) study employing the metabolic inhibitor itraconazole have been…”
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AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial by Mendell, Jerry R, Muntoni, Francesco, McDonald, Craig M, Mercuri, Eugenio M, Ciafaloni, Emma, Komaki, Hirofumi, Leon-Astudillo, Carmen, Nascimento, Andrés, Proud, Crystal, Schara-Schmidt, Ulrike, Veerapandiyan, Aravindhan, Zaidman, Craig M, Guridi, Maitea, Murphy, Alexander P, Reid, Carol, Wandel, Christoph, Asher, Damon R, Darton, Eddie, Mason, Stefanie, Potter, Rachael A, Singh, Teji, Zhang, Wenfei, Fontoura, Paulo, Elkins, Jacob S, Rodino-Klapac, Louise R

“…Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by pathogenic variants in the DMD gene that result in the absence of…”
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