يعرض 1 - 10 نتائج من 113 نتيجة بحث عن '"Douglas M Sproule"', وقت الاستعلام: 0.99s تنقيح النتائج
  1. 1
    دورية أكاديمية
    SMA-MAP: a plasma protein panel for spinal muscular atrophy.

    المؤلفون: 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

    المصدر: PLoS ONE, Vol 8, Iss 4, p e60113 (2013)

    مصطلحات موضوعية: Medicine, Science

    وصف الملف: electronic resource

    العلاقة: http://europepmc.org/articles/PMC3615018?pdf=renderTest; https://doaj.org/toc/1932-6203Test

    الوصول الحر: https://doaj.org/article/bde3060c6f104867b0d25e27fac0f7aeTest

    View record in DOAJ النص الكامل

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  2. 2
    دورية أكاديمية
    An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

    المؤلفون: Rebecca Dean, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Thomas Wiesner, Daniel C. Malone, Matthias Bischof, Walter Toro, Omar Dabbous

    المصدر: Journal of Market Access & Health Policy, Vol 9, Iss 1 (2021)

    مصطلحات موضوعية: cost-effectiveness analysis, gene therapy, spinal muscular atrophy, onasemnogene abeparvovec, cost-utility analysis, nusinersen, Public aspects of medicine, RA1-1270, Business, HF5001-6182

    العلاقة: http://dx.doi.org/10.1080/20016689.2021.1889841Test; https://doaj.org/toc/2001-6689Test; https://doaj.org/article/4ff5f47c0a944bf891888597b331385cTest

    الإتاحة: https://doi.org/10.1080/20016689.2021.1889841Test
    https://doaj.org/article/4ff5f47c0a944bf891888597b331385cTest

    View record in BASE النص الكامل

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  3. 3
    دورية أكاديمية
    Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients

    المؤلفون: Daniel C. Malone, Rebecca Dean, Ramesh Arjunji, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Omar Dabbous

    المصدر: Journal of Market Access & Health Policy, Vol 7, Iss 1 (2019)

    مصطلحات موضوعية: avxs-101, cost-effectiveness, gene-replacement therapy, nusinersen, onasemnogene abeparvovec, spinal muscular atrophy type 1, Public aspects of medicine, RA1-1270, Business, HF5001-6182

    وصف الملف: electronic resource

    العلاقة: https://doaj.org/toc/2001-6689Test

    الوصول الحر: https://doaj.org/article/930a9177e655400d90828ef1a4362fd9Test

    View record in DOAJ النص الكامل

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  4. 4
    Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue

    المؤلفون: 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

    المصدر: Nature Medicine. 27:1701-1711

    مصطلحات موضوعية: Mutation, Pathology, medicine.medical_specialty, Messenger RNA, business.industry, Genetic enhancement, Central nervous system, General Medicine, Spinal muscular atrophy, SMN1, Motor neuron, medicine.disease, medicine.disease_cause, General Biochemistry, Genetics and Molecular Biology, medicine.anatomical_structure, Systemic administration, medicine, business

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::ef6f0fd234d81724758ac7b79e4e9847Test
    https://doi.org/10.1038/s41591-021-01483-7Test


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  5. 5
    Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

    المؤلفون: 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

    المصدر: Paediatrics Publications

    مصطلحات موضوعية: Duchenne muscular dystrophy, medicine.medical_specialty, nonsense mutation Duchenne muscular dystrophy, Prednisolone, Population, Nonsense mutation, Placebo, chemistry.chemical_compound, Pregnenediones, Prednisone, Internal medicine, medicine, Humans, education, deflazacort, Retrospective Studies, education.field_of_study, business.industry, Health Policy, prednisolone, medicine.disease, Ataluren, Muscular Dystrophy, Duchenne, Deflazacort, meta-analysis, chemistry, Codon, Nonsense, prednisone, business, medicine.drug

    وصف الملف: application/pdf

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1e773db882a9b68ac18148646fe6a8d7Test
    https://ir.lib.uwo.ca/context/paedpub/article/3206/viewcontent/843.pdfTest


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  6. 6
    Response to: Alfred Sandrock, Wildon Farwell. Letter to the Editor, Comparisons Between Separately Conducted Clinical Trials: Letter to the Editor Regarding Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guérin A, et al. Adv Ther (2019) 36(5):1164-76. doi:10.1007/s12325-019-00923-8

    المؤلفون: 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

    المصدر: Advances in Therapy

    مصطلحات موضوعية: Onasemnogene abeparvovec-xioi, medicine.medical_specialty, Letter, Letter to the editor, business.industry, Pharmacology toxicology, Oligonucleotides, MEDLINE, Infant, General Medicine, Spinal muscular atrophy, NNT, Muscular Atrophy, Spinal, Clinical trial, Nusinersen, Family medicine, medicine, Humans, AVXS-101, Pharmacology (medical), business

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6ef95c92834f6943987e3169245a13dcTest
    https://doi.org/10.1007/s12325-019-01088-0Test


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  7. 7
    Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy

    المؤلفون: 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

    المصدر: Pediatric Neurology. 98:39-45

    مصطلحات موضوعية: Male, Pediatrics, medicine.medical_specialty, Genetic Vectors, Motor Disorders, Spinal Muscular Atrophies of Childhood, Sitting, Severity of Illness Index, Motor function, 03 medical and health sciences, 0302 clinical medicine, Developmental Neuroscience, 030225 pediatrics, Outcome Assessment, Health Care, medicine, Humans, Dosing, Newborn screening, business.industry, Gene replacement therapy, Age Factors, Infant, SMN Complex Proteins, Mean age, Genetic Therapy, Spinal muscular atrophy, Dependovirus, medicine.disease, SMA, Neurology, Pediatrics, Perinatology and Child Health, Female, Neurology (clinical), business, 030217 neurology & neurosurgery, Follow-Up Studies

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::91189814492917c474701ff1f3c07dcaTest
    https://doi.org/10.1016/j.pediatrneurol.2019.05.005Test


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  8. 8
    Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1

    المؤلفون: 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

    المصدر: Advances in Therapy

    مصطلحات موضوعية: 030213 general clinical medicine, medicine.medical_specialty, Sitting, 03 medical and health sciences, 0302 clinical medicine, Nusinersen, Internal medicine, Onasemnogene abeparvovec, AVXS-101, Medicine, Pharmacology (medical), Original Research, business.industry, General Medicine, Spinal muscular atrophy, medicine.disease, SMA, Confidence interval, Clinical trial, 030220 oncology & carcinogenesis, Relative risk, Number needed to treat, Indirect treatment comparison, Spinal muscular atrophy type 1 (SMA type 1), business, Neuroscience

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e36535eccac12d04ad591848174ebad0Test
    https://doi.org/10.1007/s12325-019-00923-8Test


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  9. 9
    An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

    المؤلفون: 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

    المصدر: Journal of Market Access & Health Policy, Vol 9, Iss 1 (2021)
    Journal of Market Access & Health Policy
    article-version (VoR) Version of Record

    مصطلحات موضوعية: medicine.medical_specialty, Cost–utility analysis, HF5001-6182, business.industry, onasemnogene abeparvovec, cost-effectiveness analysis, cost-utility analysis, nusinersen, Spinal muscular atrophy, Cost-effectiveness analysis, medicine.disease, gene therapy, Physical medicine and rehabilitation, Cost utility, Medicine, Business, Nusinersen, Original Research Article, Public aspects of medicine, RA1-1270, business, Research Article, spinal muscular atrophy

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::897d6b522c3e810f3d6e608db8b4c94eTest
    https://doi.org/10.1080/20016689.2021.1889841Test


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  10. 10
    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, phase 3 trial

    المؤلفون: 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

    المصدر: The Lancet. Neurology. 20(4)

    مصطلحات موضوعية: 0301 basic medicine, Male, medicine.medical_specialty, Recombinant Fusion Proteins, Population, Physical examination, SMN1, Spinal Muscular Atrophies of Childhood, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, Medicine, Humans, education, education.field_of_study, Biological Products, medicine.diagnostic_test, business.industry, Infant, Spinal muscular atrophy, Genetic Therapy, medicine.disease, Clinical trial, Survival of Motor Neuron 2 Protein, 030104 developmental biology, Clinical research, Treatment Outcome, Concomitant, Child, Preschool, Cohort, Female, Neurology (clinical), business, 030217 neurology & neurosurgery

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::21a75136d5a074e1de00eddad203b3c8Test
    https://pubmed.ncbi.nlm.nih.gov/33743228Test


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