Zobrazeno 1 - 10
of 105
pro vyhledávání: '"Douglas M Sproule"'
Autor:
Dione T Kobayashi, Jing Shi, Laurie Stephen, Karri L Ballard, Ruth Dewey, James Mapes, Brett Chung, Kathleen McCarthy, Kathryn J Swoboda, Thomas O Crawford, Rebecca Li, Thomas Plasterer, Cynthia Joyce, Biomarkers for Spinal Muscular Atrophy Study Group, Wendy K Chung, Petra Kaufmann, Basil T Darras, Richard S Finkel, Douglas M Sproule, William B Martens, Michael P McDermott, Darryl C De Vivo, Pediatric Neuromuscular Clinical Research Network, Michael G Walker, Karen S Chen
Publikováno v:
PLoS ONE, Vol 8, Iss 4, p e60113 (2013)
OBJECTIVESSpinal Muscular Atrophy (SMA) presents challenges in (i) monitoring disease activity and predicting progression, (ii) designing trials that allow rapid assessment of candidate therapies, and (iii) understanding molecular causes and conseque
Externí odkaz:
https://doaj.org/article/bde3060c6f104867b0d25e27fac0f7ae
Autor:
Daniel C. Malone, Rebecca Dean, Ramesh Arjunji, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Omar Dabbous
Publikováno v:
Journal of Market Access & Health Policy, Vol 7, Iss 1 (2019)
Background: Spinal muscular atrophy type 1 (SMA1) is a devastating genetic disease for which gene-replacement therapy may bring substantial survival and quality of life benefits. Objective: This study investigated the cost-effectiveness of onasemnoge
Externí odkaz:
https://doaj.org/article/930a9177e655400d90828ef1a4362fd9
Autor:
Arthur H.M. Burghes, Caroline Hsieh, Jerry R. Mendell, Douglas M. Sproule, Binh T. T. Chu, Kevin D. Foust, Douglas E. Feltner, Wendy K. Chung, Francesco Muntoni, Mariacristina Scoto, Christopher R. Pierson, Miriam R Conces, Stephanie Perry, Gretchen Thomsen, Petra Kaufmann, Robert F. Hevner, Basam Barkho, Janet Do, Vicki L. McGovern
Publikováno v:
Nature Medicine. 27:1701-1711
Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec (formerly AVXS-101) is a gene therapy that restores SMN production via on
Autor:
Perry B Shieh, Gary Elfring, Panayiota Trifillis, Claudio Santos, Stuart W Peltz, Julie A Parsons, Susan Apkon, Basil T Darras, Craig Campbell, Craig M McDonald, Richard J Barohn, Enrico Bertini, Kate Bushby, Brigitte Chabrol, Emma Ciafaloni, Jaume Columer, Giacomi Pietro Comi, Anne Connolly, Richard S Finkel, Kevin M Flanigan, Nathalie Goemans, Michela Guglieri, Susan T Iannaccone, Kristi J Jones, Petra Kaufmann, Janbernd Kirschner, Jean K Mah, Katherine Mathews, Eugenio Mercuri, Francesco Muntoni, Yoram Nevo, Andrés Nascimento Osorio, Yann Péréon, Rosaline Quinlivan, J. Ben Renfroe, Barry Russman, Monique Ryan, Jacinda Sampson, Ulrike Schara, Kathryn Selby, Thomas Sejersen, Douglas M Sproule, H. Lee Sweeney, Már Tulinius, Juan J Vilchez, Giuseppe Vita, Thomas Voit, Stephanie Burns-Wechsler, Brenda Wong, Ted Abresch, Erik K Henricson, Kim Coleman, Michelle Eagle, Julaine Florence, Ed Gappmaier, Craig McDonald, Hoda Z Abdel-Hamid, Clemens Bloetzer, Russell J Butterfield, Jong-Hee Chae, Jahannaz Dastgir, Isabelle Desguerre, Raul G Escobar, Erika Finanger, Peter Heydemann, Imelda Hughes, Anna Kaminska, Peter Karachunski, Martin Kudr, Timothy Lotze, Alexandra Prufer de Queiroz Campos Araujo, Maria Bernadete Dutra de Resende, Gihan Tennekoon, Haluk Topaloglu, Ricardo Erazo Torricelli, Lindsay N Alfano, Meredith K James, Linda Lowes, Anna Mayhew, Elena S Mazzone, Leslie Nelson, Kristy J Rose
Publikováno v:
Paediatrics Publications
Aim: Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Materials & methods: Placebo data from Phase I
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1e773db882a9b68ac18148646fe6a8d7
https://ir.lib.uwo.ca/context/paedpub/article/3206/viewcontent/843.pdf
https://ir.lib.uwo.ca/context/paedpub/article/3206/viewcontent/843.pdf
Autor:
Maria Lorenzi, Jeroen P. Jansen, O. Dabbous, Benit Maru, Annie Guerin, Douglas M. Sproule, Douglas E. Feltner, Ramesh Arjunji, Irina Pivneva, Eric Q. Wu, Martin Cloutier
Publikováno v:
Advances in Therapy
Autor:
Sukumar Nagendran, Markus McColly, John T. Kissel, Kelly J. Lehman, Lindsay N. Alfano, Linda Lowes, Kathleen Church, W. David Arnold, Samiah Al-Zaidy, Melissa Menier, Douglas M. Sproule, Courtney Wells, Douglas E. Feltner, Jerry R. Mendell, Thomas W. Prior, Richard Shell
Publikováno v:
Pediatric Neurology. 98:39-45
This study characterizes motor function responses after early dosing of AVXS-101 (onasemnogene abeparvovec) in gene replacement therapy in infants with severe spinal muscular atrophy type 1 (SMA1).This study is a follow-up analysis of 12 infants with
Autor:
Douglas E. Feltner, Irina Pivneva, Martin Cloutier, Eric Q. Wu, Ramesh Arjunji, O. Dabbous, Annie Guerin, Douglas M. Sproule, Benit Maru, Jeroen P. Jansen, Maria Lorenzi
Publikováno v:
Advances in Therapy
Introduction Infants with spinal muscular atrophy (SMA) type 1 typically face a decline in motor function and a severely shortened life expectancy. Clinical trials for SMA type 1 therapies, onasemnogene abeparvovec (AVXS-101) and nusinersen, demonstr
Autor:
Phil Cyr, Matthias Bischof, Daniel C. Malone, Ivar Jensen, Benit Maru, Thomas Wiesner, Rebecca Dean, Walter Toro, Beckley Miller, Douglas E. Feltner, O. Dabbous, Douglas M. Sproule
Publikováno v:
Journal of Market Access & Health Policy, Vol 9, Iss 1 (2021)
Journal of Market Access & Health Policy
article-version (VoR) Version of Record
Journal of Market Access & Health Policy
article-version (VoR) Version of Record
Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results. Objective: To compare the manufacturer’s proprietary
Autor:
Jerry R. Mendell, Meredith Schultz, Douglas M. Sproule, Haojun Ouyang, Claudia A. Chiriboga, Loren D M Pena, John W. Day, Craig M. Zaidman, Anne M. Connolly, Susan T. Iannaccone, Thomas O. Crawford, Basil T. Darras, Nancy L. Kuntz, Deepa H. Chand, Edward C. Smith, Thomas A Macek, Perry B. Shieh, Sitra Tauscher-Wisniewski, Douglas E. Feltner, Richard S. Finkel, Jennifer M. Kwon
Publikováno v:
The Lancet. Neurology. 20(4)
Summary Background Spinal muscular atrophy type 1 is a motor neuron disorder resulting in death or the need for permanent ventilation by age 2 years. We aimed to evaluate the safety and efficacy of onasemnogene abeparvovec (previously known as AVXS-1
Autor:
Thomas A. Macek, Edward C. Smith, John W. Day, Perry B. Shieh, S.T. Iannaconne, H. Ouyang, Anne M. Connolly, Richard Shell, Craig M. Zaidman, J. Mendell, Douglas M. Sproule, Thomas O. Crawford, Sitra Tauscher-Wisniewski, Claudia A. Chiriboga, Meredith Schultz, L.D.M. Peña, Richard S. Finkel, Basil T. Darras, Francis G. Ogrinc, P. Thomasma, Jennifer M. Kwon, Douglas E. Feltner, Nancy L. Kuntz, Elaine Kernbauer
Publikováno v:
C96. BEST OF PEDIATRICS.