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1. First Results from the REAL-time Transient Acquisition backend (REALTA) at the Irish LOFAR station

Author

Murphy, P. C., Callanan, P., McCauley, J., McKenna, D. J., Fionnagáin, D. Ó, Louis, C. K., Redman, M. P., Cañizares, L. A., Carley, E. P., Maloney, S. A., Coghlan, B., Daly, M., Scully, J., Dooley, J., Gajjar, V., Giese, C., Brennan, A., Keane, E. F., Maguire, C. A., Quinn, J., Mooney, S., Ryan, A. M., Walsh, J., Jackman, C. M., Golden, A., Ray, T. P., Doyle, J. G., Rigney, J., Burton, M., and Gallagher, P. T.

Subjects
Astrophysics - Instrumentation and Methods for Astrophysics
Abstract

Modern radio interferometers such as the LOw Frequency ARray (LOFAR) are capable of producing data at hundreds of gigabits to terabits per second. This high data rate makes the analysis of radio data cumbersome and computationally expensive. While high performance computing facilities exist for large national and international facilities, that may not be the case for instruments operated by a single institution or a small consortium. Data rates for next generation radio telescopes are set to eclipse those currently in operation, hence local processing of data will become all the more important. Here, we introduce the REAL-time Transient Acquisition backend (REALTA), a computing backend at the Irish LOFAR station (I-LOFAR) which facilitates the recording of data in near real-time and post-processing. We also present first searches and scientific results of a number of radio phenomena observed by I-LOFAR and REALTA, including pulsars, fast radio bursts (FRBs), rotating radio transients (RRATs), the search for extraterrestrial intelligence (SETI), Jupiter, and the Sun., Comment: 12 pages, 10 figures, accepted for publication in Astronomical instrumentation section of Astronomy & Astrophysics 24/08/2021

Published
2021
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2. WFIRST-2.4: What Every Astronomer Should Know

Author

Spergel, D., Gehrels, N., Breckinridge, J., Donahue, M., Dressler, A., Gaudi, B. S., Greene, T., Guyon, O., Hirata, C., Kalirai, J., Kasdin, N. J., Moos, W., Perlmutter, S., Postman, M., Rauscher, B., Rhodes, J., Wang, Y., Weinberg, D., Centrella, J., Traub, W., Baltay, C., Colbert, J., Bennett, D., Kiessling, A., Macintosh, B., Merten, J., Mortonson, M., Penny, M., Rozo, E., Savransky, D., Stapelfeldt, K., Zu, Y., Baker, C., Cheng, E., Content, D., Dooley, J., Foote, M., Goullioud, R., Grady, K., Jackson, C., Kruk, J., Levine, M., Melton, M., Peddie, C., Ruffa, J., and Shaklan, S.

Subjects
Astrophysics - Instrumentation and Methods for Astrophysics
Abstract

The Astro2010 Decadal Survey recommended a Wide Field Infrared Survey Telescope (WFIRST) as its top priority for a new large space mission. The report of the WFIRST-AFTA Science Definition Team (SDT) presents a Design Reference Mission for WFIRST that employs one of the 2.4-m, Hubble-quality mirror assemblies recently made available to NASA. The 2.4-m primary mirror enables a mission with greater sensitivity and higher angular resolution than the smaller aperture designs previously considered for WFIRST, increasing both the science return of the primary surveys and the capabilities of WFIRST as a Guest Observer facility. The option of adding an on-axis, coronagraphic instrument would enable imaging and spectroscopic studies of planets around nearby stars. This short article, produced as a companion to the SDT report, summarizes the key points of the WFIRST-2.4 DRM. It highlights the remarkable opportunity that the 2.4-m telescope affords for advances in many fields of astrophysics and cosmology, including dark energy, the demographics and characterization of exoplanets, the evolution of galaxies and quasars, and the stellar populations of the Milky Way and its neighbors., Comment: 15 pages, 9 figures, 1 table, Companion article to the SDT report, arXiv:1305.5422, added pointer to WFIRST-AFTA SDT report and corrected line color description in Figure 2 caption

Published
2013

3. Wide-Field InfraRed Survey Telescope-Astrophysics Focused Telescope Assets WFIRST-AFTA Final Report

Author

Spergel, D., Gehrels, N., Breckinridge, J., Donahue, M., Dressler, A., Gaudi, B. S., Greene, T., Guyon, O., Hirata, C., Kalirai, J., Kasdin, N. J., Moos, W., Perlmutter, S., Postman, M., Rauscher, B., Rhodes, J., Wang, Y., Weinberg, D., Centrella, J., Traub, W., Baltay, C., Colbert, J., Bennett, D., Kiessling, A., Macintosh, B., Merten, J., Mortonson, M., Penny, M., Rozo, E., Savransky, D., Stapelfeldt, K., Zu, Y., Baker, C., Cheng, E., Content, D., Dooley, J., Foote, M., Goullioud, R., Grady, K., Jackson, C., Kruk, J., Levine, M., Melton, M., Peddie, C., Ruffa, J., and Shaklan, S.

Subjects
Astrophysics - Instrumentation and Methods for Astrophysics
Abstract

The Astro2010 Decadal Survey recommended a Wide Field Infrared Survey Telescope (WFIRST) as its top priority for a new large space mission. As conceived by the decadal survey, WFIRST would carry out a dark energy science program, a microlensing program to determine the demographics of exoplanets, and a general observing program utilizing its ultra wide field. In October 2012, NASA chartered a Science Definition Team (SDT) to produce, in collaboration with the WFIRST Project Office at GSFC and the Program Office at JPL, a Design Reference Mission (DRM) for an implementation of WFIRST using one of the 2.4-m, Hubble-quality mirror assemblies recently made available to NASA. This DRM builds on the work of the earlier WFIRST SDT, reported by Green et al. (2012). The 2.4-m primary mirror enables a mission with greater sensitivity and higher angular resolution than the 1.3-m and 1.1-m designs considered previously, increasing both the science return of the primary surveys and the capabilities of WFIRST as a Guest Observer facility. The option of adding an on-axis, coronagraphic instrument would enable imaging and spectroscopic studies of planets around nearby stars. This document presents the final report of the SDT., Comment: 190 pages, 118 figures, 15 tables, For a short summary of the report highlights, see arXiv:1305.5425, added pointer to the summary of this report and corrected line labels in the caption of Figure 2-2

Published
2013

7. Cognitive Underpinnings of Moral Reasoning in Adolescence: The Contribution of Executive Functions

Author

Vera-Estay, E., Dooley, J. J., and Beauchamp, M. H.

Abstract

Adolescence is a developmental period characterized by intense changes, which impact the interaction between individuals and their environments. Moral reasoning (MR) is an important skill during adolescence because it guides social decisions between right and wrong. Identifying the cognitive underpinnings of MR is essential to understanding the development of this function. The aim of this study was to explore predictors of MR in typically developing adolescents (n = 92, 33 males, M = 16.3 years, SD = 2.2 years) and the specific contribution of higher order cognitive processing using an innovative visual MR assessment tool and measures of executive functioning and intelligence. MR maturity was correlated with four executive functions (cognitive flexibility, feedback utilization, conceptual reasoning, verbal fluency) and was predicted by four variables: age, intelligence, nonverbal flexibility and verbal fluency. Overall, these results contribute to a better understanding of MR during adolescence and highlight the importance of using innovative tools to measure social cognition.

Published
2015
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18. Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta‐analysis of disease progression rates in recent multicenter clinical trials

Author

McDonald CM, A238., Sajeev, G, Yao, Z, Mcdonnell, E, Elfring, G, Souza, M, Peltz, Sw, Darras, Bt, Shieh, Pb, Cox, Da, Landry, J, Signorovitch, J, Campbell, C, Torricelli, Re, Finkel, Rs, Flanigan, Km, Goemans, N, Heydemann, P, Kaminska, A, Kirschner, J, Muntoni, F, Osorio, An, Schara, U, Sejersen, T, Sweeney, Hl, Topaloglu, H, Tulinius, M, Vilchez, Jj, Voit, T, Wong, B, Alfano, Ln, Eagle, M, James, Mk, Lowes, L, Mayhew, A, Mazzone, Es, Nelson, L, Rose, Kj, Abdel-Hamid, Hz, Apkon, Sd, Barohn, Rj, Bertini, E, Bloetzer, C, de Vaud LC, Butterfield, Rj, Chabrol, B, Chae, Jh, Jongno-Gu, Dr, Comi, Gp, Dastgir, J, Desguerre, I, Escobar, Rg, Finanger, E, Guglieri, M, Hughes, I, Iannaccone, St, Jones, Kj, Karachunski, P, Kudr, M, Lotze, T, Mah, Jk, Mathews, K, Nevo, Y, Parsons, J, Péréon, Y, de Queiroz Campos Araujo AP, Renfroe, Jb, de Mbd, R, Ryan, M, Selby, K, Tennekoon, G, Vita, G, Abdel-Hamid, H, Apkon, S, Barohn, R, Belousova, E, Brandsema, J, Bruno, C, Burnette, W, Butterfield, R, Byrne, B, Carlo, J, Chandratre, S, Comi, G, Connolly, A, De Groot, I, Deconinck, N, Dooley, J, Dubrovsky, A, Durigneux, J, Finkel, R, Frank, Lm, Harper, A, Hattori, A, Herguner, O, Iannaccone, S, Janas, J, Jong, Yj, Komaki, H, Kuntz, N, Lee, Wt, Leung, E, Mah, J, Cm, M, Mercuri, E, Mcmillan, H, Mueller-Felber, W, Lopez de Munain, A, Nakamura, A, Niks, E, Ogata, K, Pascual, S, Pegoraro, E, Pereon, Y, Renfroe, B, Sanka, Rb, Schallner, J, Sendra, Ii, Servais, L, Smith, E, Sparks, S, Victor, R, Wicklund, M, Wilichoswki, E, and Wong, B.

Subjects
Male, Duchenne muscular dystrophy, 0301 basic medicine, medicine.medical_specialty, Physiology, medicine.drug_class, Prednisolone, Anti-Inflammatory Agents, Walking, 030105 genetics & heredity, Placebo, prednisone/prednisolone, ambulatory function, deflazacort, meta-analysis, 03 medical and health sciences, Cellular and Molecular Neuroscience, 0302 clinical medicine, Pregnenediones, Prednisone, Physiology (medical), Internal medicine, Humans, Multicenter Studies as Topic, Medicine, Child, Clinical Research Articles, Randomized Controlled Trials as Topic, Clinical Research Article, business.industry, medicine.disease, Confidence interval, Muscular Dystrophy, Duchenne, Deflazacort, Treatment Outcome, meta‐analysis, Ambulatory, Disease Progression, Corticosteroid, Neurology (clinical), business, 030217 neurology & neurosurgery, medicine.drug
Abstract

Introduction In this study we characterized disease progression over 48 weeks among boys receiving deflazacort vs prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. Methods Ambulatory boys with DMD receiving placebo in the phase 3 ataluren (N = 115) and tadalafil (N = 116) trials were included. The trials required at least 6 months of prior corticosteroid use and stable baseline dosing. Associations between corticosteroid use and 48‐week changes in ambulatory function were estimated using mixed models. Adjusted differences between corticosteroid groups were pooled in a meta‐analysis. Results In the meta‐analysis, deflazacort‐treated patients vs prednisone/prednisolone‐treated patients experienced, on average, lower declines of 28.3 meters on 6‐minute walk distance (95% confidence interval [CI], 5.7, 50.9; 2.9 seconds on rise from supine [95% CI, 0.9, 4.9 seconds]; 2.3 seconds on 4‐stair climb [95% CI, 0.5, 4.1 seconds]; and 2.9 [95% CI, 0.1, 5.8] points on the North Star Ambulatory Assessment linearized score). Discussion Deflazacort‐treated patients experienced significantly lower functional decline over 48 weeks.

Published
2019

19. Letters

Author

Crosby, Joe H., Dooley, J. K., Dickson, Tom, and Rockefeller, Laurance S.

Published
2007

20. Science Goals and Mission Architecture of the Europa Lander Mission Concept

Author

Hand, K. P., primary, Phillips, C. B., additional, Murray, A., additional, Garvin, J. B., additional, Maize, E. H., additional, Gibbs, R. G., additional, Reeves, G., additional, Martin, A. M. San, additional, Tan-Wang, G. H., additional, Krajewski, J., additional, Hurst, K., additional, Crum, R., additional, Kennedy, B. A., additional, McElrath, T. P., additional, Gallon, J. C., additional, Sabahi, D., additional, Thurman, S. W., additional, Goldstein, B., additional, Estabrook, P., additional, Lee, S. W., additional, Dooley, J. A., additional, Brinckerhoff, W. B., additional, Edgett, K. S., additional, German, C. R., additional, Hoehler, T. M., additional, Hörst, S. M., additional, Lunine, J. I., additional, Paranicas, C., additional, Nealson, K., additional, Smith, D. E., additional, Templeton, A. S., additional, Russell, M. J., additional, Schmidt, B., additional, Christner, B., additional, Ehlmann, B., additional, Hayes, A., additional, Rhoden, A., additional, Willis, P., additional, Yingst, R. A., additional, Craft, K., additional, Cameron, M. E., additional, Nordheim, T., additional, Pitesky, J., additional, Scully, J., additional, Hofgartner, J., additional, Sell, S. W., additional, Barltrop, K. J., additional, Izraelevitz, J., additional, Brandon, E. J., additional, Seong, J., additional, Jones, J.-P., additional, Pasalic, J., additional, Billings, K. J., additional, Ruiz, J. P., additional, Bugga, R. V., additional, Graham, D., additional, Arenas, L. A., additional, Takeyama, D., additional, Drummond, M., additional, Aghazarian, H., additional, Andersen, A. J., additional, Andersen, K. B., additional, Anderson, E. W., additional, Babuscia, A., additional, Backes, P. G., additional, Bailey, E. S., additional, Balentine, D., additional, Ballard, C. G., additional, Berisford, D. F., additional, Bhandari, P., additional, Blackwood, K., additional, Bolotin, G. S., additional, Bovre, E. A., additional, Bowkett, J., additional, Boykins, K. T., additional, Bramble, M. S., additional, Brice, T. M., additional, Briggs, P., additional, Brinkman, A. P., additional, Brooks, S. M., additional, Buffington, B. B., additional, Burns, B., additional, Cable, M. L., additional, Campagnola, S., additional, Cangahuala, L. A., additional, Carr, G. A, additional, Casani, J. R., additional, Chahat, N. E., additional, Chamberlain-Simon, B. K., additional, Cheng, Y., additional, Chien, S. A., additional, Cook, B. T., additional, Cooper, M., additional, DiNicola, M., additional, Clement, B., additional, Dean, Z., additional, Cullimore, E. A., additional, Curtis, A. G., additional, Croix, J-P. de la, additional, Pasquale, P. Di, additional, Dodd, E. M., additional, Dubord, L. A., additional, Edlund, J. A., additional, Ellyin, R., additional, Emanuel, B., additional, Foster, J. T., additional, Ganino, A. J., additional, Garner, G. J., additional, Gibson, M. T., additional, Gildner, M., additional, Glazebrook, K. J., additional, Greco, M. E., additional, Green, W. M., additional, Hatch, S. J., additional, Hetzel, M. M., additional, Hoey, W. A., additional, Hofmann, A. E., additional, Ionasescu, R., additional, Jain, A., additional, Jasper, J. D., additional, Johannesen, J. R., additional, Johnson, G. K., additional, Jun, I., additional, Katake, A. B., additional, Kim-Castet, S. Y., additional, Kim, D. I., additional, Kim, W., additional, Klonicki, E. F., additional, Kobeissi, B., additional, Kobie, B. D., additional, Kochocki, J., additional, Kokorowski, M., additional, Kosberg, J. A., additional, Kriechbaum, K., additional, Kulkarni, T. P., additional, Lam, R. L., additional, Landau, D. F., additional, Lattimore, M. A., additional, Laubach, S. L., additional, Lawler, C. R., additional, Lim, G., additional, Lin, J. Y, additional, Litwin, T. E., additional, Lo, M. W., additional, Logan, C. A., additional, Maghasoudi, E., additional, Mandrake, L., additional, Marchetti, Y., additional, Marteau, E., additional, Maxwell, K. A., additional, Namee, J. B. Mc, additional, Mcintyre, O., additional, Meacham, M., additional, Melko, J. P., additional, Mueller, J., additional, Muliere, D. A., additional, Mysore, A., additional, Nash, J., additional, Ono, H., additional, Parker, J. M., additional, Perkins, R. C., additional, Petropoulos, A. E, additional, Gaut, A., additional, Gomez, M. Y. Piette, additional, Casillas, R. P., additional, Preudhomme, M., additional, Pyrzak, G., additional, Rapinchuk, J., additional, Ratliff, J. M., additional, Ray, T. L., additional, Roberts, E. T., additional, Roffo, K., additional, Roth, D. C., additional, Russino, J. A., additional, Schmidt, T. M., additional, Schoppers, M. J., additional, Senent, J. S., additional, Serricchio, F., additional, Sheldon, D. J., additional, Shiraishi, L. R., additional, Shirvanian, J., additional, Siegel, K. J., additional, Singh, G., additional, Sirota, A. R., additional, Skulsky, E. D., additional, Stehly, J. S., additional, Strange, N. J., additional, Stevens, S. U., additional, Sunada, E. T., additional, Tepsuporn, S. P., additional, Tosi, L. P. C., additional, Trawny, N., additional, Uchenik, I., additional, Verma, V., additional, Volpe, R. A., additional, Wagner, C. T., additional, Wang, D., additional, Willson, R. G., additional, Wolff, J. L., additional, Wong, A. T., additional, Zimmer, A. K., additional, Sukhatme, K. G., additional, Bago, K. A., additional, Chen, Y., additional, Deardorff, A. M., additional, Kuch, R. S., additional, Lim, C., additional, Syvertson, M. L., additional, Arakaki, G. A., additional, Avila, A., additional, DeBruin, K. J., additional, Frick, A., additional, Harris, J. R., additional, Heverly, M. C., additional, Kawata, J. M., additional, Kim, S.-K., additional, Kipp, D. M., additional, Murphy, J., additional, Smith, M. W., additional, Spaulding, M. D., additional, Thakker, R., additional, Warner, N. Z., additional, Yahnker, C. R., additional, Young, M. E., additional, Magner, T., additional, Adams, D., additional, Bedini, P., additional, Mehr, L., additional, Sheldon, C., additional, Vernon, S., additional, Bailey, V., additional, Briere, M., additional, Butler, M., additional, Davis, A., additional, Ensor, S., additional, Gannon, M., additional, Haapala-Chalk, A., additional, Hartka, T., additional, Holdridge, M., additional, Hong, A., additional, Hunt, J., additional, Iskow, J., additional, Kahler, F., additional, Murray, K., additional, Napolillo, D., additional, Norkus, M., additional, Pfisterer, R., additional, Porter, J., additional, Roth, D., additional, Schwartz, P., additional, Wolfarth, L., additional, Cardiff, E. H., additional, Grob, E. W., additional, Adam, J. R., additional, Betts, E., additional, Norwood, J., additional, Heller, M. M., additional, Voskuilen, T., additional, Sakievich, P., additional, Gray, L., additional, Hansen, D. J., additional, Irick, K. W., additional, Hewson, J. C., additional, Lamb, J., additional, Stacy, S. C., additional, Brotherton, C. M., additional, Tappan, A. S, additional, Benally, D., additional, Thigpen, H., additional, Ortiz, E., additional, Sandoval, D., additional, Ison, A. M., additional, Warren, M., additional, Stromberg, P. G., additional, Thelen, P. M., additional, Blasy, B., additional, Nandy, P., additional, Haddad, A. W., additional, Trujillo, L. B., additional, Wiseley, T. H., additional, Bell, S. A., additional, Teske, N. P., additional, Post, C., additional, Torres-Castro, L., additional, Grosso, C., additional, and Wasiolek, M., additional

Published
2022
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23. First results from the REAL-time Transient Acquisition backend (REALTA) at the Irish LOFAR station

Author

Murphy, P. C., primary, Callanan, P., additional, McCauley, J., additional, McKenna, D. J., additional, Fionnagáin, D. Ó, additional, Louis, C. K., additional, Redman, M. P., additional, Cañizares, L. A., additional, Carley, E. P., additional, Maloney, S. A., additional, Coghlan, B., additional, Daly, M., additional, Scully, J., additional, Dooley, J., additional, Gajjar, V., additional, Giese, C., additional, Brennan, A., additional, Keane, E. F., additional, Maguire, C. A., additional, Quinn, J., additional, Mooney, S., additional, Ryan, A. M., additional, Walsh, J., additional, Jackman, C. M., additional, Golden, A., additional, Ray, T. P., additional, Doyle, J. G., additional, Rigney, J., additional, Burton, M., additional, and Gallagher, P. T., additional

Published
2021
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27. Rural and remote obstetric care close to home: program description, evaluation and discussion of Sioux Lookout Meno Ya Win Health Centre obstetrics

Author

Dooley, J., Kelly, Len, Pierre-Hansen, N. St, Antone, I., O'Driscoll, T., and Guilfoyle J.

Subjects
Indigenous peoples -- Health aspects, Indigenous peoples -- Demographic aspects, Indigenous peoples -- Research, Obstetrical nursing -- Health aspects, Obstetrical nursing -- Services, Family medicine -- Health aspects, Family medicine -- Demographic aspects, Family medicine -- Research
Abstract

Problem being addressed: Aboriginal and non-Aboriginal women in rural and remote settings struggle to access obstetric care close to home. Objective of the program: To deliver a full range of modern and safe obstetric care to 28 remote Aboriginal communities served by rural-based health care. Program description: Rural family physicians provide intrapartum, cesarean delivery and anesthesia services to 350 rural, primarily Aboriginal women in a collegial, supportive environment. Conclusion: Rural and remote obstetric services need support before they fail. Patient volume, remote location and organizational culture are key elements. Evidence teaches us that outcomes are best when women deliver closer to home. Probleme aborde : Les femmes autochtones et non autochtones vivant en region rurale et eloignee ont du mal a obtenir des soins obstetricaux pres de chez elles. Objectif du programme : Offrir une gamme complete de soins obstetricaux modernes et securitaires a 28 communautes autochtones eloignees, desservies par des services de soins de sante ruraux. Description du programme : Prestation de services intrapartum, de cesariennes et d'anesthesie obstetricale par des medecins de famille exercant en milieu rural, a 350 femmes, surtout autochtones, dans un contexte de collegialite et d'entraide. Conclusion : Il faut soutenir les services obstetricaux en region rurale et eloignee, faute de quoi ils pourraient disparaitre. Le volume de patientes, l'eloignement geographique et la culture organisationnelle sont des elements cles dont on doit tenir compte. L'experience nous enseigne que l'issue des grossesses est meilleure lorsque les femmes peuvent accoucher plus pres de leur lieu de residence., INTRODUCTION Delivery of obstetric services to remote communities in Canada will always be challenging. Despite decades of experience there are limited descriptions of successful models of care. In 1997, the [...]

Published
2009

28. Elongational viscosity of LDPEs and polystyrenes using entrance loss data

Author

Walczak, K., Gupta, M., Koppi, K.A., Dooley, J., and Spalding, M.A.

Subjects
Rheometers -- Usage, Finite element method -- Models -- Measurement -- Chemical properties -- Usage, Polystyrene -- Chemical properties -- Usage, Viscosity -- Measurement -- Chemical properties -- Usage -- Models, Engineering and manufacturing industries, Science and technology, Usage, Chemical properties, Models, Measurement
Abstract

For two low-density polyethylenes and two polystyrenes, axisymmetric and planar elongational viscosities are estimated using entrance loss data from capillary and slit rheometers, respectively. The elongational viscosity is estimated by optimizing the values of various parameters in the Sarkar-Gupta elongational viscosity model such that the entrance loss predicted by a finite element simulation agrees with the corresponding experimental data. The predicted entrance loss is in good agreement with the experimental data at high flow rates. The difference in the experimental and predicted entrance loss at lower flow rates might have been caused by large error in the experimental data in this range. POLYM. ENG. SCI., 48:223-232, 2008. © 2007 Society of Plastics Engineers, INTRODUCTION Owing to their long chain molecules, polymers exhibit very complex rheological behavior. Some of the phenomena due to this macromolecular structure of polymers such as die swell and rod [...]

Published
2008

32. Setting the stage for first fringes with the Magdalena Ridge Observatory Interferometer

Author

Creech-Eakman, Michelle J., primary, Romero, V. D., additional, Haniff, Christopher A., additional, Buscher, David F., additional, Young, J. S., additional, Olivares, Andres, additional, Ligon, E. R., additional, Payne, I., additional, Avila, Bastian, additional, Blevins, Sterling, additional, Chowdhury, Naseef, additional, Collins, Robert, additional, Dahl, Chuck, additional, Das Roy, Dipanjan, additional, Dooley, J., additional, Etscorn, Dylan, additional, Etscorn, Shelbi, additional, Farris, Allen, additional, Fisher, Martin, additional, Garcia, Erica, additional, Gabaldon, Mateo, additional, Gino, Colleen, additional, Halder, Ratna, additional, Hosseini, Omid, additional, Jaynes, Brandon, additional, Jencka, Louis, additional, Johnston, Perry, additional, Jojola, Stephen, additional, Luis, James, additional, Maes, Jennie, additional, Martin, William, additional, Matthews, Tyler, additional, McKeen, Cailyn, additional, Mohammadi, Sina, additional, Mortimer, Dan, additional, Norouzi, Siavash, additional, Ochoa, Danielle, additional, Panta, Bhola, additional, Pino, Juan, additional, Salayandia, Isaac, additional, Salcido, Chris, additional, Sanchez, Amanda, additional, Santoro, Fernando, additional, Seneta, E. Bodie, additional, Sun, X., additional, Wilson, Donald, additional, and Wolfram, Jeanette, additional

Published
2020
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38. Royal academy of medicine in Ireland section of biomedical sciences: Proceedings of meeting held Thursday & Friday 26th, 27th June, 1997 in University of Limerick

Author

MacDonncha, C., Watson, A. W. S., Jakeman, P. M., Welsh, L. J., McGrath, S., Watson, A. W. S., Ooi, H., Brady, H. R., O’Regan, R. G, McLoughlin, P., Cantillon, D., Bradford, A., Deacy, C., Garrett, M., Lowe, A. S., Baxter, G. D., Walker, M. D., Hirst, D. G., Kennovin, G. D., Leek, M., Watson, A. W. S., MacDonncha, C., Watson, A. W. S., Watson, A. W. S., Robinson, J., Hill, I. D., Baxter, G. D., Allen, J. M., Black, A. J., Baxter, G. D., Hughes, S., Bell, A. L., Orr, G., McDonagh, S., Baxter, G. D., Baker, R., Cadogan, E., Hopkins, N., O’Regan, R. G., McLoughlin, P., Lynch, F., O’Regan, R. G., McLoughlin, P., Nolan, Y. M., Connor, T. J., Kelly, J. P., Leonard, E., Shen, Y., Redmond, A. M., Kelly, J. P., Leonard, B. E., Connor, T. J., McNamara, M. G., Finn, D., Currid, A., O’Malley, M., Redmond, A. M., Kelly, J. P., Leonard, B. E., Healy, D. G., Kelly, J. P., Leonard, B. E., Walker, M. D., Lowe, A. S., Hirst, D. G., Kennovin, G. D., Leek, M., Cullen, V. C., Wilson, S., Love, G., Keenan, A. K., O’Hagan, B., Murray, D., Dooley, J. S. G., McKerr, G., Allen, J. M., Doyle, P., Healy, D. G., Kelly, J. P., Leonard, B. E., Hanna-Mitchell, A. T., Gebruers, E. M., Hill, I. D., Robinson, J., Baxter, G. D., Allen, J. M., Leonard, M., Ryan, M. P., Healy, E., Keane, T., Healy, E., Ryan, M. P., Egan, D., Dempsey, M., Healy, E., Ryan, M. P., Kelleher, S., Keenan, A. K., Esfandiary, H., Gilmore, W. S., Allen, J. M., Hannigan, B. M., Al-Assar, O., Robson, T., McKeown, S. R., and Hirst, D. G.

Published
1998
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46. Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

Author

Conrado, D. J., Larkindale, J., Berg, A., Hill, M., Burton, J., Abrams, K. R., Abresch, R. T., Bronson, A., Chapman, D., Crowther, M., Duong, T., Gordish-Dressman, H., Harnisch, L., Henricson, E., Kim, S., Mcdonald, C. M., Schmidt, S., Vong, C., Wang, X., Wong, B. L., Yong, F., Romero, K., Vishwanathan, V., Chidambaranathan, S., Douglas Biggar, W., Mcadam, L. C., Mah, J. K., Tulinius, M., Cnaan, A., Morgenroth, L. P., Leshner, R., Tesi-Rocha, C., Thangarajh, M., Kornberg, A., Ryan, M., Nevo, Y., Dubrovsky, A., Clemens, P. R., Abdel-Hamid, H., Connolly, A. M., Pestronk, A., Teasley, J., Bertorini, T. E., Webster, R., Kolski, H., Kuntz, N., Driscoll, S., Bodensteiner, J. B., Gorni, K., Lotze, T., Day, J. W., Karachunski, P., Henricson, E. K., Joyce, N. C., Campbell, C., Torricelli, R. E., Finkel, R. S., Flanigan, K. M., Goemans, N., Heydemann, P., Kaminska, A., Kirschner, J., Muntoni, F., Osorio, A. N., Schara, U., Sejersen, T., Shieh, P. B., Sweeney, H. L., Topaloglu, H., Vilchez, J. J., Voit, T., Wong, B., Alfano, L. N., Eagle, M., James, M. K., Lowes, L., Mayhew, A., Mazzone, E. S., Nelson, L., Rose, K. J., Abdel-Hamid, H. Z., Apkon, S. D., Barohn, R. J., Bertini, E., Bloetzer, C., Devaud, L. C., Butterfield, R. J., Chabrol, B., Chae, J. H., Jongno-Gu, D. R., Comi, G. P., Darras, B. T., Dastgir, J., Desguerre, I., Escobar, R. G., Finanger, E., Guglieri, M., Hughes, I., Iannaccone, S. T., Jones, K. J., Kudr, M., Mathews, K., Parsons, J., Pereon, Y., de Queiroz Campos Araujo, A. P., Renfroe, J. B., de Resende, M. B. D., Selby, K., Tennekoon, G., Vita, G., Apkon, S., Barohn, R., Belousova, E., Brandsema, J., Bruno, C., Burnette, W., Butterfield, R., Byrne, B., Carlo, J., Chandratre, S., Comi, G., Connolly, A., De Groot I, I., Deconinck, N., Dooley, J., Durigneux, J., Finkel, R., Frank, L. M., Harper, A., Hattori, A., Herguner, O., Iannaccone, S., Janas, J., Jong, Y. J., Komaki, H., Lee, W. T., Leung, E., Mah, J., Mercuri, E., Mcmillan, H., Mueller-Felber, W., de Munain A, L., Nakamura, A., Niks, E., Ogata, K., Pascual, S., Pegoraro, E., Renfroe, B., Sanka, R. B., Schallner, J., Sendra, I. I., Servais, L., Smith, E., Sparks, S., Victor, R., Wicklund, M., Wilichoswki, E., Carter, G. T., and Servais, LJP

Subjects
Orphan Drug Production, Duchenne muscular dystrophy, Pharmacy, Model-informed drug development, 030226 pharmacology & pharmacy, Models, Biological, Food and drug administration, 03 medical and health sciences, 0302 clinical medicine, Models, medicine, Humans, Regulatory science, Computer Simulation, Muscular Dystrophy, Drug development tools, Duchenne muscular dystrophy consortium (D-RSC), Rare diseases, Regulatory endorsement, Clinical Trials as Topic, Muscular Dystrophy, Duchenne, United States, United States Food and Drug Administration, Pharmacology, Protocol (science), business.industry, Duchenne, Biological, medicine.disease, Clinical trial, Risk analysis (engineering), Drug development, 030220 oncology & carcinogenesis, Aggregate data, Business
Abstract

Drug development for rare diseases is challenged by small populations and limited data. This makes development of clinical trial protocols difficult and contributes to the uncertainty around whether or not a potential therapy is efficacious. The use of data standards to aggregate data from multiple sources, and the use of such integrated databases to develop statistical models can inform protocol development and reduce the risks in developing new therapies. Achieving regulatory endorsement of such models through defined pathways at the US Food and Drug Administration and European Medicines Authority allows such tools to be used by the drug development community for defined contexts of use without further need for discussion of the underlying model(s). The Duchenne Regulatory Science Consortium (D-RSC) has brought together multiple stakeholders to develop a clinical trial simulation tool for Duchenne muscular dystrophy using such an approach. Here we describe the work of D-RSC as an example of how such an approach may be effective at reducing uncertainty in drug development for rare diseases, and thus bringing effective therapies to patients faster.

Published
2019

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