Observational study of spinal muscular atrophy type I and implications for clinical trials
Finkel, Richard S, McDermott, Michael P, Kaufmann, Petra, Darras, Basil T, Chung, Wendy K, Sproule, Douglas M, Kang, Peter B, Foley, A Reghan, Yang, Michelle L, Martens, William B, Oskoui, Maryam, Glanzman, Allan M, Flickinger, Jean, Montes, Jacqueline, Dunaway, Sally, O'Hagen, Jessica, Quigley, Janet, Riley, Susan, Benton, Maryjane, Ryan, Patricia A, Montgomery, Megan, Marra, Jonathan, Gooch, Clifton, De Vivo, Darryl C
Published in Neurology (26.08.2014)
Published in Neurology (26.08.2014)
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Prospective cohort study of spinal muscular atrophy types 2 and 3
Kaufmann, Petra, McDermott, Michael P, Darras, Basil T, Finkel, Richard S, Sproule, Douglas M, Kang, Peter B, Oskoui, Maryam, Constantinescu, Andrei, Gooch, Clifton L, Foley, A Reghan, Yang, Michele L, Tawil, Rabi, Chung, Wendy K, Martens, William B, Montes, Jacqueline, Battista, Vanessa, O'Hagen, Jessica, Dunaway, Sally, Flickinger, Jean, Quigley, Janet, Riley, Susan, Glanzman, Allan M, Benton, Maryjane, Ryan, Patricia A, Punyanitya, Mark, Montgomery, Megan J, Marra, Jonathan, Koo, Benjamin, De Vivo, Darryl C
Published in Neurology (30.10.2012)
Published in Neurology (30.10.2012)
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Journal Article
Octopamine stabilizes conduction reliability of an unmyelinated axon during hypoxic stress
Money, T G A, Sproule, M K J, Cross, K P, Robertson, R M
Published in Journal of neurophysiology (01.09.2016)
Published in Journal of neurophysiology (01.09.2016)
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Journal Article
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R., Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Nagendran, Sukumar, L’Italien, James, Sproule, Douglas M., Wells, Courtney, Cardenas, Jessica A., Heitzer, Marjet D., Kaspar, Allan, Corcoran, Sarah, Braun, Lyndsey, Likhite, Shibi, Miranda, Carlos, Meyer, Kathrin, Foust, K.D., Burghes, Arthur H.M., Kaspar, Brian K.
Published in The New England journal of medicine (02.11.2017)
Published in The New England journal of medicine (02.11.2017)
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Journal Article
Natural history of MELAS associated with mitochondrial DNA m.3243A>G genotype
Kaufmann, P, Engelstad, K, Wei, Y, Kulikova, R, Oskoui, M, Sproule, D M, Battista, V, Koenigsberger, D Y, Pascual, J M, Shanske, S, Sano, M, Mao, X, Hirano, M, Shungu, D C, Dimauro, S, De Vivo, D C
Published in Neurology (29.11.2011)
Published in Neurology (29.11.2011)
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Journal Article
Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients
Malone, Daniel C., Dean, Rebecca, Arjunji, Ramesh, Jensen, Ivar, Cyr, Phil, Miller, Beckley, Maru, Benit, Sproule, Douglas M., Feltner, Douglas E., Dabbous, Omar
Published in Journal of market access & health policy (2019)
Published in Journal of market access & health policy (2019)
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Journal Article
Giant axonal neuropathy: An updated perspective on its pathology and pathogenesis
Johnson-Kerner, Bethany L., Roth, Lisa, Greene, J. Palmer, Wichterle, Hynek, Sproule, Douglas M.
Published in Muscle & nerve (01.10.2014)
Published in Muscle & nerve (01.10.2014)
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Journal Article
High resolution computed tomographic assessment of airway wall thickness in chronic asthma: reproducibility and relationship with lung function and severity
Little, S A, Sproule, M W, Cowan, M D, Macleod, K J, Robertson, M, Love, J G, Chalmers, G W, McSharry, C P, Thomson, N C
Published in Thorax (01.03.2002)
Published in Thorax (01.03.2002)
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Journal Article
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)
Dean, Rebecca, Jensen, Ivar, Cyr, Phil, Miller, Beckley, Maru, Benit, Sproule, Douglas M., Feltner, Douglas E., Wiesner, Thomas, Malone, Daniel C., Bischof, Matthias, Toro, Walter, Dabbous, Omar
Published in Journal of market access & health policy (2021)
Published in Journal of market access & health policy (2021)
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Journal Article
Toxicity and Efficacy of Stereotactic Ablative Body Radiotherapy for Moderately Central Non-small Cell Lung Cancers Using 50 Gy in Five Fractions
Rulach, R., McLoone, P., Lumsden, G., McKay, S., MacLaren, V., Macphee, J., Moore, K., Omand, M., Sproule, M., Currie, S., Aitken, A., Ferguson, R., Valentine, R., Houston, P., Harrow, S., Hicks, J.
Published in Clinical oncology (Royal College of Radiologists (Great Britain)) (01.04.2020)
Published in Clinical oncology (Royal College of Radiologists (Great Britain)) (01.04.2020)
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Journal Article
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, Jerry R, Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R, Prior, Thomas W, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T, Nagendran, Sukumar, L’Italien, James, Sproule, Douglas M, Wells, Courtney, Cardenas, Jessica A, Heitzer, Marjet D, Kaspar, Allan, Corcoran, Sarah, Braun, Lyndsey, Likhite, Shibi, Miranda, Carlos, Meyer, Kathrin, Foust, K.D, Burghes, Arthur H.M, Kaspar, Brian K
Published in The New England journal of medicine (02.11.2017)
Published in The New England journal of medicine (02.11.2017)
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Journal Article
Therapeutic developments in spinal muscular atrophy
Sproule, Douglas M., Kaufmann, Petra
Published in Therapeutic Advances in Neurological Disorders (01.05.2010)
Published in Therapeutic Advances in Neurological Disorders (01.05.2010)
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Book Review
Journal Article
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
Day, John W, Finkel, Richard S, Chiriboga, Claudia A, Connolly, Anne M, Crawford, Thomas O, Darras, Basil T, Iannaccone, Susan T, Kuntz, Nancy L, Peña, Loren D M, Shieh, Perry B, Smith, Edward C, Kwon, Jennifer M, Zaidman, Craig M, Schultz, Meredith, Feltner, Douglas E, Tauscher-Wisniewski, Sitra, Ouyang, Haojun, Chand, Deepa H, Sproule, Douglas M, Macek, Thomas A, Mendell, Jerry R
Published in Lancet neurology (01.04.2021)
Published in Lancet neurology (01.04.2021)
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Journal Article
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
Lowes, Linda P., Alfano, Lindsay N., Arnold, W. David, Shell, Richard, Prior, Thomas W., McColly, Markus, Lehman, Kelly J., Church, Kathleen, Sproule, Douglas M., Nagendran, Sukumar, Menier, Melissa, Feltner, Douglas E., Wells, Courtney, Kissel, John T., Al-Zaidy, Samiah, Mendell, Jerry
Published in Pediatric neurology (01.09.2019)
Published in Pediatric neurology (01.09.2019)
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Journal Article
Mitochondrial Encephalopathy, Lactic Acidosis, and Strokelike Episodes
Sproule, Douglas M., Kaufmann, Petra
Published in Annals of the New York Academy of Sciences (01.10.2008)
Published in Annals of the New York Academy of Sciences (01.10.2008)
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Journal Article
Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
Al‐Zaidy, Samiah, Pickard, A. Simon, Kotha, Kavitha, Alfano, Lindsay N., Lowes, Linda, Paul, Grace, Church, Kathleen, Lehman, Kelly, Sproule, Douglas M., Dabbous, Omar, Maru, Benit, Berry, Katherine, Arnold, W. David, Kissel, John T., Mendell, Jerry R., Shell, Richard
Published in Pediatric pulmonology (01.02.2019)
Published in Pediatric pulmonology (01.02.2019)
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Journal Article
Thigh muscle volume measured by magnetic resonance imaging is stable over a 6-month interval in spinal muscular atrophy
Sproule, Douglas M, Montgomery, Megan J, Punyanitya, Mark, Shen, Wei, Dashnaw, Stephen, Montes, Jacqueline, Dunaway, Sally, Finkel, Richard, Darras, Basil, Vivo, Darryl C De, Kaufmann, Petra
Published in Journal of child neurology (01.10.2011)
Published in Journal of child neurology (01.10.2011)
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