Histone deacetylases suppress CGG repeat-induced neurodegeneration via transcriptional silencing in models of fragile X tremor ataxia syndrome von Todd, Peter K, Oh, Seok Yoon, Krans, Amy, Pandey, Udai B, Di Prospero, Nicholas A, Min, Kyung-Tai, Taylor, J Paul, Paulson, Henry L

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Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints von Meyer, Elias Laurin, Mesenbrink, Peter, Di Prospero, Nicholas A, Pericàs, Juan M, Glimm, Ekkehard, Ratziu, Vlad, Sena, Elena, König, Franz

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Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons von Pericàs, Juan M., Tacke, Frank, Anstee, Quentin M., Di Prospero, Nicholas A., Kjær, Mette Skalshøj, Mesenbrink, Peter, Koenig, Franz, Genescà, Joan, Ratziu, Vlad

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Review article: The need for more efficient and patient‐oriented drug development pathways in NASH—setting the scene for platform trials von Pericàs, Juan M., Di Prospero, Nicholas A., Anstee, Quentin M., Mesenbrinck, Peter, Kjær, Mette S., Rivera‐Esteban, Jesús, Koenig, Franz, Sena, Elena, Pais, Raluca, Manzano, Ramiro, Genescà, Joan, Tacke, Frank, Ratziu, Vlad, DiProspero, Nicholas A., Ratziu, Vlad, Pericàs, Juan M., Kjær, Mette S., Anstee, Quentin M., Tacke, Frank, Mesenbrinck, Peter, Rivera‐Esteban, Jesús, Koenig, Franz, Sena, Elena, Manzano, Ramiro, Genescà, Joan, Pais, Raluca, Kara, Leila, Meyer, Elias, Duca, Anna, Kline, Timothy, Aaes‐Jørgensen, Anders, Balthaus, Tania, Preville, Natalie, Zhang, Lingjiao, Capuano, George, Morello, Salvatore, Mielke, Tobias, Penna, Sabina H., Posch, Martin

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Neurological effects of high-dose idebenone in patients with Friedreich's ataxia: a randomised, placebo-controlled trial von Di Prospero, Nicholas A, MD, Baker, Angela, BSN, Jeffries, Neal, PhD, Fischbeck, Kenneth H, MD

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Clinical features of spinal and bulbar muscular atrophy von Rhodes, Lindsay E., Freeman, Brandi K., Auh, Sungyoung, Kokkinis, Angela D., La Pean, Alison, Chen, Cheunju, Lehky, Tanya J., Shrader, Joseph A., Levy, Ellen W., Harris-Love, Michael, Di Prospero, Nicholas A., Fischbeck, Kenneth H.

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Altered gene expression and DNA damage in peripheral blood cells from Friedreich's ataxia patients: cellular model of pathology von Haugen, Astrid C, Di Prospero, Nicholas A, Parker, Joel S, Fannin, Rick D, Chou, Jeff, Meyer, Joel N, Halweg, Christopher, Collins, Jennifer B, Durr, Alexandra, Fischbeck, Kenneth, Van Houten, Bennett

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Effects of rifampin, cyclosporine A, and probenecid on the pharmacokinetic profile of canagliflozin, a sodium glucose co-transporter 2 inhibitor, in healthy participants von Devineni, Damayanthi, Vaccaro, Nicole, Murphy, Joe, Curtin, Christopher, Mamidi, Rao N V S, Weiner, Sveta, Wang, Shean-Sheng, Ariyawansa, Jay, Stieltjes, Hans, Wajs, Ewa, Di Prospero, Nicholas A, Rothenberg, Paul

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Impaired mitochondrial function in psychiatric disorders von Manji, Husseini, Kato, Tadafumi, Di Prospero, Nicholas A., Ness, Seth, Beal, M. Flint, Krams, Michael, Chen, Guang

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Mechanism of Increased LDL (Low-Density Lipoprotein) and Decreased Triglycerides With SGLT2 (Sodium-Glucose Cotransporter 2) Inhibition von Basu, Debapriya, Huggins, Lesley-Ann, Scerbo, Diego, Obunike, Joseph, Mullick, Adam E, Rothenberg, Paul L, Di Prospero, Nicholas A, Eckel, Robert H, Goldberg, Ira J

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Expression of expanded polyglutamine targets profilin for degradation and alters actin dynamics von Burnett, Barrington G, Andrews, Jaime, Ranganathan, Srikanth, Fischbeck, Kenneth H, Di Prospero, Nicholas A

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Standard and modified statistical mune evaluations in spinal-bulbar muscular atrophy von Lehky, Tanya J., Chen, Cheun Ju, di Prospero, Nicholas A., Rhodes, Lindsay E., Fischbeck, Kenneth, Floeter, Mary Kay

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Efficacy and safety of glucagon‐like peptide‐1/glucagon receptor co‐agonist JNJ‐64565111 in individuals with type 2 diabetes mellitus and obesity: A randomized dose‐ranging study... von Di Prospero, Nicholas A., Yee, Jaqueline, Frustaci, Mary E., Samtani, Mahesh N., Alba, Maria, Fleck, Penny

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Biochemical and Phenotypic Abnormalities in Kynurenine Aminotransferase II-Deficient Mice von Yu, Ping, Di Prospero, Nicholas A., Sapko, Michael T., Cai, Tao, Chen, Amy, Melendez-Ferro, Miguel, Du, Fu, Whetsell, William O., Guidetti, Paolo, Schwarcz, Robert, Tagle, Danilo A.

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Exercise Capacity and Idebenone Intervention in Children and Adolescents With Friedreich Ataxia von Drinkard, Bart E., MSPT, Keyser, Randall E., PhD, Paul, Scott M., MD, Arena, Ross, PhD, PT, Plehn, Jonathan F., MD, Yanovski, Jack A., MD, PhD, Di Prospero, Nicholas A., MD, PhD

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Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy von Avila, Amy M, Burnett, Barrington G, Taye, Addis A, Gabanella, Francesca, Knight, Melanie A, Hartenstein, Parvana, Cizman, Ziga, Di Prospero, Nicholas A, Pellizzoni, Livio, Fischbeck, Kenneth H, Sumner, Charlotte J

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Pharmacokinetics and pharmacodynamics of canagliflozin in pediatric patients with type 2 diabetes von Tamborlane, William V, Polidori, David, Argenti, Domenick, Di Prospero, Nicholas A

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Assessing Function and Endurance in Adults with Spinal and Bulbar Muscular Atrophy: Validity of the Adult Myopathy Assessment Tool von Levy, Ellen, Davenport, Todd E., Di Prospero, Nicholas A., Fischbeck, Kenneth H., Chen, Cheunju, Auh, Sungyoung, La Pean Kirschner, Alison, Kokkinis, Angela, Shrader, Joseph A., Joe, Galen, Fernandez-Rhodes, Lindsay, Harris-Love, Michael O., Li, Li

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The Subclinical Cardiomyopathy of Friedreich's Ataxia in a Pediatric Population von Plehn, Jonathan F., Hasbani, Keren, Ernst, Inez, Horton, Kenneth D., Drinkard, Bart E., Di Prospero, Nicholas A.

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Therapeutics development for triplet repeat expansion diseases von Di Prospero, Nicholas A, Fischbeck, Kenneth H

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