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3. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial

Author

Dimachkie, Mazen, Statland, Jeffrey, Pasnoor, Mamatha, Jawdat, Omar, Heim, Andrew, Ciersdorff, Ali, Sasidharan, Sandhya, Currence, Melissa, Levine, Todd, Otutoa, Rebecca, Cooper, Angelina, Mozaffar, Tahseen, Habib, Ali, Cauchi, Jonathan, Ung, Shannon, Mathew, Veena, Hernandez, Isela, Gibson, Summer, Bromberg, Mark, Mahoney, Kyle, Neate, Crystal, Janecki, Teresa, Papadakis, Mike, Freimer, Miriam, Kaschalk, MacKenzie, Heintzman, Sarah, Wicklund, Matthew, Baines, Brenna, Vareldzis, Alexa, Hyslop, Emily, Blume, Brianna, Ciafaloni, Emma, Luebbe, Elizabeth, Eichinger, Katy, Martens, William, Gregory, Stephanie, Janciuras, Joanne, Amato, Anthony, Doughty, Christopher, Roe, Kristen, Flynn, Patricia, Russo, Emily, Lloyd, Thomas, Albayda, Jemima, Tiniakou, Eleni, Thomas, Simone, Jones, Sarah, Solorzano, Guillermo, Elliott, Matthew, Burns, Ted, Crowell, Allison, Eggleston, Deborah, Wagoner, Mary, Shaibani, Aziz, Oates, Chantae, Machado, Pedro, Hanna, Michael, Greensmith, Linda, Ahmed, Mhoriam, Vivekanandam, Vinojini, Appleby, Matthew, Ransley, George, Eshun, Edwin Eshun, Skorupinska, Iwona, Germain, Louise, Laxa, Ana Marie, Pontes, Joana Roca, Bellin, Anna, Anifowoshe, Dolapo, Machado, Pedro M, McDermott, Michael P, Blaettler, Thomas, Sundgreen, Claus, Amato, Anthony A, Gibson, Summer B, Jones, Sarah M, Levine, Todd D, Lloyd, Thomas E, Shaibani, Aziz I, Rosholm, Anders, Carstensen, Tim Dehli, Bonefeld, Karen, Jørgensen, Anders Nørkær, Phonekeo, Karina, Heim, Andrew J, Herbelin, Laura, Barohn, Richard J, Hanna, Michael G, and Dimachkie, Mazen M

Published
2023
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4. Lean tissue mass measurements by dual-energy X-ray absorptiometry and associations with strength and functional outcome measures in facioscapulohumeral muscular dystrophy

Author

Wang, Leo H., Leung, Doris G., Wagner, Kathryn R., Lowry, Sarah J., McDermott, Michael P., Eichinger, Katy, Higgs, Kiley, Walker, Michaela, Lewis, Leann, Martens, William B., Mul, Karlien, Sansone, Valeria A., Shieh, Perry, Elsheikh, Bakri, LoRusso, Samantha, Butterfield, Russell J., Johnson, Nicholas, Preston, Matthew R., Messina, Carmelo, Carraro, Elena, Tawil, Rabi, and Statland, Jeff

Published
2023
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5. A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.

Author

Crow, Rebecca, Hart, Kimberly, McDermott, Michael, Tawil, Rabi, Martens, William, Herr, Barbara, McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy, Eagle, Michele, Brown, Mary, Hirtz, Deborah, Lochmuller, Hanns, Straub, Volker, Ciafaloni, Emma, Shieh, Perry, Spinty, Stefan, Childs, Anne-Marie, Manzur, Adnan, Morandi, Lucia, Butterfield, Russell, Horrocks, Iain, Roper, Helen, Flanigan, Kevin, Kuntz, Nancy, Mah, Jean, Morrison, Leslie, Darras, Basil, von der Hagen, Maja, Schara, Ulrike, Wilichowski, Ekkehard, Mongini, Tiziana, Vita, Giuseppe, Barohn, Richard, Finkel, Richard, Wicklund, Matthew, McMillan, Hugh, Hughes, Imelda, Pegoraro, Elena, Bryan Burnette, W, Howard, James, Thangarajh, Mathula, Campbell, Craig, Griggs, Robert, Bushby, Kate, Guglieri, Michela, and McDonald, Craig

Subjects
Academic-led clinical trial, Clinical trial, Clinical trial regulations, Duchenne muscular dystrophy, Rare disease, Budgets, Checklist, Clinical Trials as Topic, Contracts, Humans, International Cooperation, Multicenter Studies as Topic, Muscular Dystrophy, Duchenne, Patient Selection, Rare Diseases, Research Design, Research Support as Topic, Steroids, Time Factors, Treatment Outcome
Abstract

BACKGROUND: Trials in rare diseases have many challenges, among which are the need to set up multiple sites in different countries to achieve recruitment targets and the divergent landscape of clinical trial regulations in those countries. Over the past years, there have been initiatives to facilitate the process of international study set-up, but the fruits of these deliberations require time to be operationally in place. FOR-DMD (Finding the Optimum Steroid Regimen for Duchenne Muscular Dystrophy) is an academic-led clinical trial which aims to find the optimum steroid regimen for Duchenne muscular dystrophy, funded by the National Institutes of Health (NIH) for 5 years (July 2010 to June 2015), anticipating that all sites (40 across the USA, Canada, the UK, Germany and Italy) would be open to recruitment from July 2011. However, study start-up was significantly delayed and recruitment did not start until January 2013. METHOD: The FOR-DMD study is used as an example to identify systematic problems in the set-up of international, multi-centre clinical trials. The full timeline of the FOR-DMD study, from funding approval to site activation, was collated and reviewed. Systematic issues were identified and grouped into (1) study set-up, e.g. drug procurement; (2) country set-up, e.g. competent authority applications; and (3) site set-up, e.g. contracts, to identify the main causes of delay and suggest areas where anticipatory action could overcome these obstacles in future studies. RESULTS: Time from the first contact to site activation across countries ranged from 6 to 24 months. Reasons of delay were universal (sponsor agreement, drug procurement, budgetary constraints), country specific (complexity and diversity of regulatory processes, indemnity requirements) and site specific (contracting and approvals). The main identified obstacles included (1) issues related to drug supply, (2) NIH requirements regarding contracting with non-US sites, (3) differing regulatory requirements in the five participating countries, (4) lack of national harmonisation with contracting and the requirement to negotiate terms and contract individually with each site and (5) diversity of languages needed for study materials. Additionally, as with many academic-led studies, the FOR-DMD study did not have access to the infrastructure and expertise that a contracted research organisation could provide, organisations often employed in pharmaceutical-sponsored studies. This delay impacted recruitment, challenged the clinical relevance of the study outcomes and potentially delayed the delivery of the best treatment to patients. CONCLUSION: Based on the FOR-DMD experience, and as an interim solution, we have devised a checklist of steps to not only anticipate and minimise delays in academic international trial initiation but also identify obstacles that will require a concerted effort on the part of many stakeholders to mitigate.

Published
2018

6. Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

Author

Straub, Volker, Childs, Anne-Marie, Ciafaloni, Emma, Shieh, Perry B., Spinty, Stefan, Butterfield, Russell J., Horrocks, Iain, Roper, Helen, Maggi, Lorenzo, Baranello, Giovanni, Flanigan, Kevin M., Kuntz, Nancy L., Manzur, Adnan Y., Darras, Basil T., Kang, Peter, Mah, Jean K., Mongini, Tiziana, Ricci, Federica, Morrison, Leslie, Krzesniak-Swinarska, Monika, von der Hagen, Maja, Finkel, Richard S., Kumar, Ashutosh, Wicklund, Matthew, McDonald, Craig M., Henricson, Erik K., Schara-Schmidt, Ulrike, Wilichowski, Ekkehard, Barohn, Richard J., Statland, Jeffrey, Kirschner, Janbernd, Vita, Giuseppe, Vita, Gian Luca, Howard, James F., Jr., Hughes, Imelda, McMillan, Hugh J., Pegoraro, Elena, Bello, Luca, Burnette, W. Bryan, Thangarajh, Mathula, Chang, Taeun, Campbell, Craig, McColl, Elaine, McDermott, Michael P., Martens, William B., Guglieri, Michela, and Griggs, Robert C.

Published
2021
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7. Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.

Author

Guglieri, Michela, Bushby, Kate, McDermott, Michael, Hart, Kimberly, Tawil, Rabi, Martens, William, Herr, Barbara, McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy, Eagle, Michele, Brown, Mary, Willis, Tracey, Hirtz, Deborah, Shieh, Perry, Straub, Volker, Childs, Anne-Marie, Ciafaloni, Emma, Butterfield, Russell, Horrocks, Iain, Spinty, Stefan, Flanigan, Kevin, Kuntz, Nancy, Baranello, Giovanni, Roper, Helen, Morrison, Leslie, Mah, Jean, Manzur, Adnan, Schara, Ulrike, von der Hagen, Maja, Barohn, Richard, Campbell, Craig, Darras, Basil, Finkel, Richard, Vita, Giuseppe, Hughes, Imelda, Mongini, Tiziana, Pegoraro, Elena, Wicklund, Matthew, Wilichowski, Ekkehard, Bryan Burnette, W, Howard, James, McMillan, Hugh, Thangarajh, Mathula, Griggs, Robert, and McDonald, Craig

Subjects
Deflazacort, Duchenne muscular dystrophy, Prednisolone, Randomized, Standards of care, Child, Child, Preschool, Disability Evaluation, Double-Blind Method, Drug Administration Schedule, Heart Function Tests, Humans, Immunosuppressive Agents, Male, Muscle Strength, Muscular Dystrophy, Duchenne, Patient Satisfaction, Prednisone, Pregnenediones, Range of Motion, Articular, Research Design, Vital Capacity
Abstract

Despite corticosteroids being the only treatment documented to improve strength and function in boys with Duchenne muscular dystrophy (DMD) corticosteroid prescription is inconsistent and in some countries, corticosteroids are not prescribed. We are conducting a clinical trial that (1) compares the 3 most frequently prescribed corticosteroid regimes; (2) standardizes treatment of DMD complications; and (3) standardizes prevention of corticosteroid side effects. Investigators at 38 sites in 5 countries plan to recruit 300 boys aged 4-7 who are randomly assigned to one of three regimens: daily prednisone; daily deflazacort; or intermittent prednisone (10days on/10days off). Boys are followed for a minimum of 3years to assess the relative effectiveness and adverse event profiles of the different regimens. The primary outcome is a 3-dimensional variable consisting of log-transformed time to rise from the floor, forced vital capacity, and subject/parent satisfaction with treatment, each averaged over all post-baseline visits. The study protocol includes evidence- and consensus-based treatment of DMD complications and of corticosteroid side effects. This study seeks to establish a standard corticosteroid regimen for DMD. Since all new interventions for DMD are being developed as add-on therapies to corticosteroids, defining the optimum regimen is of importance for all new treatments.

Published
2017

8. Spatial Soundscape Superposition and Multimodal Interaction

Author

Cohen, Michael, Martens, William L., Hartmann, William M., Editor-in-Chief, Ando, Yoichi, Series Editor, Au, Whitlow W. L., Series Editor, Baggeroer, Arthur B., Series Editor, Fuller, Christopher R., Series Editor, Kuperman, William A., Series Editor, Miller, Joanne L., Series Editor, Tolstoy, Alexandra I., Series Editor, Blauert, Jens, editor, and Braasch, Jonas, editor

Published
2020
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10. Nusinersen Treatment in Adults with Spinal Muscular Atrophy

Author

Duong, Tina, Wolford, Connie, McDermott, Michael P., Macpherson, Chelsea E., Pasternak, Amy, Glanzman, Allan M., Martens, William B., Kichula, Elizabeth, Darras, Basil T., De Vivo, Darryl C., Zolkipli-Cunningham, Zarazuela, Finkel, Richard S., Zeineh, Michael, Wintermark, Max, Sampson, Jacinda, Hagerman, Katharine A., Young, Sally Dunaway, and Day, John W.

Published
2021
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13. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial

Author

Machado, Pedro M, primary, McDermott, Michael P, additional, Blaettler, Thomas, additional, Sundgreen, Claus, additional, Amato, Anthony A, additional, Ciafaloni, Emma, additional, Freimer, Miriam, additional, Gibson, Summer B, additional, Jones, Sarah M, additional, Levine, Todd D, additional, Lloyd, Thomas E, additional, Mozaffar, Tahseen, additional, Shaibani, Aziz I, additional, Wicklund, Matthew, additional, Rosholm, Anders, additional, Carstensen, Tim Dehli, additional, Bonefeld, Karen, additional, Jørgensen, Anders Nørkær, additional, Phonekeo, Karina, additional, Heim, Andrew J, additional, Herbelin, Laura, additional, Barohn, Richard J, additional, Hanna, Michael G, additional, Dimachkie, Mazen M, additional, Dimachkie, Mazen, additional, Statland, Jeffrey, additional, Pasnoor, Mamatha, additional, Jawdat, Omar, additional, Heim, Andrew, additional, Ciersdorff, Ali, additional, Sasidharan, Sandhya, additional, Currence, Melissa, additional, Levine, Todd, additional, Otutoa, Rebecca, additional, Cooper, Angelina, additional, Habib, Ali, additional, Cauchi, Jonathan, additional, Ung, Shannon, additional, Mathew, Veena, additional, Hernandez, Isela, additional, Gibson, Summer, additional, Bromberg, Mark, additional, Mahoney, Kyle, additional, Neate, Crystal, additional, Janecki, Teresa, additional, Papadakis, Mike, additional, Kaschalk, MacKenzie, additional, Heintzman, Sarah, additional, Baines, Brenna, additional, Vareldzis, Alexa, additional, Hyslop, Emily, additional, Blume, Brianna, additional, Luebbe, Elizabeth, additional, Eichinger, Katy, additional, Martens, William, additional, Gregory, Stephanie, additional, Janciuras, Joanne, additional, Amato, Anthony, additional, Doughty, Christopher, additional, Roe, Kristen, additional, Flynn, Patricia, additional, Russo, Emily, additional, Lloyd, Thomas, additional, Albayda, Jemima, additional, Tiniakou, Eleni, additional, Thomas, Simone, additional, Jones, Sarah, additional, Solorzano, Guillermo, additional, Elliott, Matthew, additional, Burns, Ted, additional, Crowell, Allison, additional, Eggleston, Deborah, additional, Wagoner, Mary, additional, Shaibani, Aziz, additional, Oates, Chantae, additional, Machado, Pedro, additional, Hanna, Michael, additional, Greensmith, Linda, additional, Ahmed, Mhoriam, additional, Vivekanandam, Vinojini, additional, Appleby, Matthew, additional, Ransley, George, additional, Eshun, Edwin Eshun, additional, Skorupinska, Iwona, additional, Germain, Louise, additional, Laxa, Ana Marie, additional, Pontes, Joana Roca, additional, Bellin, Anna, additional, and Anifowoshe, Dolapo, additional

Published
2023
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17. Mexiletine in Myotonic Dystrophy Type 1: A Randomized, Double-Blind, Placebo-Controlled Trial

Author

Heatwole, Chad, Luebbe, Elizabeth, Rosero, Spencer, Eichinger, Katy, Martens, William, Hilbert, James, Dekdebrun, Jeanne, Dilek, Nuran, Zizzi, Christine, Johnson, Nicholas, Puwanant, Araya, Tawil, Rabi, Schifitto, Giovanni, Beck, Christopher A., Richeson, J. Franklin, Zareba, Wojciech, Thornton, Charles, McDermott, Michael P., and Moxley, Richard, III

Published
2021
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18. Reference curves of motor function outcomes in young steroid‐naïve males with Duchenne muscular dystrophy.

Author

Hoskens, Jasmine, Schiava, Marianela, Goemans, Nathalie, Feys, Hilde, McDermott, Michael P., Martens, William B., Mayhew, Anna, Griggs, Robert C., Klingels, Katrijn, and Guglieri, Michela

Subjects
DUCHENNE muscular dystrophy, QUANTILE regression, MOTOR ability, MALES
Abstract

Aim: To investigate functional motor performance in a large cohort of young steroid‐naïve males with Duchenne muscular dystrophy (DMD) and typically developing males, and to develop specific reference curves for both groups. Also, to describe associations between anthropometric values and functional motor outcomes. Method: Cross‐sectional data of 196 steroid‐naïve males with DMD aged 4 to 8 years and 497 typically developing males aged 2 years 6 months to 8 years were included. Both groups were evaluated with the time to rise from the floor test, 10‐metre walk/run test, 6‐minute walk test, and North Star Ambulatory Assessment. Reference curves with centiles 5%, 10%, 25%, 50%, 75%, 90%, and 95% were estimated using quantile regression. Results: Males with DMD scored significantly worse on all functional motor outcomes than age‐matched typically developing males (p < 0.001): 89% to 95% of the males with DMD scored below the 5th centile of the typically developing males. No or weak correlations exist between anthropometric values and functional motor outcomes. Interpretation: The estimated reference curves can support consultation with families of young males with DMD and can support the evaluation of treatment for reaching motor skills and functional motor outcomes compared with typically developing males. [ABSTRACT FROM AUTHOR]

Published
2024
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20. A longitudinal study of disease progression in facioscapulohumeral muscular dystrophy (FSHD).

Author

Varma, Anika, Todinca, Michael S., Eichinger, Katy, Heininger, Susanne, Dilek, Nuran, Martens, William, Tawil, Rabi, Statland, Jeffrey, Kissel, John T., McDermott, Michael P., and Heatwole, Chad

Abstract

Introduction/Aims: In preparation for clinical trials, it is important to better understand how disease burden changes over time in facioscapulohumeral muscular dystrophy (FSHD) and to assess the capability of select metrics to detect these changes. This study aims to evaluate FSHD disease progression over 1 year and to examine the sensitivity of several outcome measures in detecting changes during this interval. Methods: We conducted a 12‐month prospective observational study of 41 participants with FSHD. Participants were evaluated at baseline, 6 months, and 12 months with serial strength testing (manual muscle testing or MMT and maximum voluntary isometric contraction testing or MVICT), functional testing (FSHD‐Composite Outcome Measure or FSHD‐COM, FSHD Clinical Severity Score or CSS, and FSHD Evaluation Score or FES), sleep and fatigue assessments, lean body mass measurements, respiratory testing, and the FSHD‐Health Index patient‐reported outcome. Changes in these outcome measures were assessed over the 12‐month period. Associations between changes in outcome measures and both age and sex were also examined. Results: In a 12‐month period, FSHD participant function remained largely stable with a mild worsening of strength, measured by MMT and standardized MVICT scores, and a mild loss in lean body mass. Discussion: The abilities and disease burden of adults with FSHD are largely static over a 12‐month period with participants demonstrating a mild average reduction in some measures of strength. Selection of patients, outcome measures, and trial duration should be carefully considered during the design and implementation of future clinical studies involving FSHD patients. [ABSTRACT FROM AUTHOR]

Published
2024
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21. The FSHD Composite Outcome Measure (FSHD-COM) is Reliable, Valid, and Measures Disease Progression (S7.005)

Author

Eichinger, Katy, primary, McDermott, Michael, additional, Higgs, Kiley, additional, Walker, Michaela, additional, Lewis, Leann, additional, Martens, William, additional, Leung, Doris, additional, Stinson, Nikia, additional, Holzer, Megan M., additional, Sacconi, Sabrina, additional, Garcia, Jeremy, additional, De Paz Benito, Victor, additional, Mul, Karlien, additional, Sansone, Valeria, additional, Carraro, Elena, additional, Becchiati, Stefano, additional, Frisoni, Maria Chiara, additional, Wang, Leo, additional, Kieu, Catherine, additional, Shieh, Perry, additional, Skura, Christy, additional, Elsheikh, Bakri, additional, Kelly, Kristina, additional, Jaworek, Andrea, additional, LoRusso, Samantha, additional, Butterfield, Russell, additional, Wilson, Amelia, additional, McIntyre, Melissa, additional, Johnson, Nicholas, additional, Butler, Amanda, additional, Jones, Aileen, additional, Hayes, Melissa, additional, Baker, Lindsay, additional, Sasidharan, Sandhya, additional, Tawil, Alrabi, additional, and Statland, Jeffrey, additional

Published
2023
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22. Motor Outcomes to Validate Evaluations in Facioscapulohumeral muscular dystrophy (MOVE FSHD): Preliminary Baseline Characteristics (S7.004)

Author

Walker, Michaela, primary, Butterfield, Russell, additional, Day, John, additional, Eichinger, Katy, additional, Elsheikh, Bakri, additional, Friedman, Seth, additional, Genge, Angela, additional, Higgs, Kiley, additional, Johnson, Nicholas, additional, Jones, Peter, additional, Leung, Doris, additional, Lewis, Leann, additional, Lochmuller, Hanns, additional, O'Ferrall, Erin, additional, Martens, William, additional, Shaw, Dennis, additional, Shieh, Perry, additional, Subramony, S, additional, Trivedi, Jaya, additional, Wang, Leo, additional, Wicklund, Matthew, additional, Tawil, Alrabi, additional, and Statland, Jeffrey, additional

Published
2023
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23. Spatial Navigation by Seated Users of Multimodal Augmented Reality Systems

Author

Martens William L. and Cohen Michael

Subjects
Social Sciences
Abstract

When seated users of multimodal augmented reality (AR) systems attempt to navigate unfamiliar environments, they can become disoriented during their initial travel through a remote environment that is displayed for them via that AR display technology. Even when the multimodal displays provide mutually coherent visual, auditory, and vestibular cues to the movement of seated users through a remote environment (such as a maze), those users may make errors in judging their own orientation and position relative to their starting point, and also may have difficulty determining what moves to make in order to return themselves to their starting point. In a number of investigations using multimodal AR systems featuring realtime servocontrolled movement of seated users, the relative contribution of spatial auditory display technology was examined across a variety of spatial navigation scenarios. The results of those investigations have implications for the effective use of the auditory component of a multimodal AR system in applications supporting spatial navigation through a physical environment.

Published
2021
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24. Applying adaptive recognition of the learner’s vowel space to English pronunciation training of native speakers of Japanese

Author

Martens William L. and Wang Rui

Subjects
Social Sciences
Abstract

When native speakers of Japanese are taught English as a second language, there are difficulties with their training in pronunciation of American English vowels that can be ameliorated though adaptive recognition of the learner’s vowel space. This paper reports on the development of an online Computer-Assisted Language Learning (CALL) environment that provides Japanese learners with customized target utterances of 12 single-syllable words that are synthesized according to an adaptive recognition of the learner’s vowel space. These customized target utterances provide each learner with examples of each of 12 American English monophthongs in consonant-vowel-consonant (CVC) context in order to sound as if they had been uttered by the learners themselves. This adaptive process was incorporated into a successfully developed tool for Computer-Assisted Pronunciation Training (CAPT) which gave more appropriate pronunciation targets to each learner, rather than forcing the learners to attempt to match the formant frequencies of their own utterances to those of the target utterances as produced by a speaker exhibiting a different vowel space (i.e., a speaker with a different vocal tract length).

Published
2021
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25. Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes

Author

Dunaway Young, Sally, primary, Pasternak, Amy, additional, Duong, Tina, additional, McGrattan, Katlyn E., additional, Stranberg, Sarah, additional, Maczek, Elizabeth, additional, Dias, Courtney, additional, Tang, Whitney, additional, Parker, Dana, additional, Levine, Alexis, additional, Rohan, Alyssa, additional, Wolford, Connie, additional, Martens, William, additional, McDermott, Michael P., additional, Darras, Basil T., additional, and Day, John W., additional

Published
2023
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26. Quantitative Evaluation of Lower Extremity Joint Contractures in Spinal Muscular Atrophy: Implications for Motor Function

Author

Salazar, Rachel, Montes, Jacqueline, Dunaway Young, Sally, McDermott, Michael P., Martens, William, Pasternak, Amy, Quigley, Janet, Mirek, Elizabeth, Glanzman, Allan M., Civitello, Matt, Gee, Richard, Duong, Tina, Mazzone, Elena S., Main, Marion, Mayhew, Anna, Ramsey, Danielle, Muni Lofra, Robert, Coratti, Giorgia, Fanelli, Lavinia, De Sanctis, Roberto, Forcina, Nicola, Chiriboga, Claudia, Darras, Basil T., Tennekoon, Gihan I., Scoto, Mariacristina, Day, John W., Finkel, Richard, Muntoni, Francesco, Mercuri, Eugenio, and De Vivo, Darryl C.

Published
2018
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27. Perception of Audio-Generated and Custom Motion Programs in Multimedia Display of Action-Oriented DVD Films

Author

Walker, Kent, Martens, William L., Hutchison, David, editor, Kanade, Takeo, editor, Kittler, Josef, editor, Kleinberg, Jon M., editor, Mattern, Friedemann, editor, Mitchell, John C., editor, Naor, Moni, editor, Nierstrasz, Oscar, editor, Pandu Rangan, C., editor, Steffen, Bernhard, editor, Sudan, Madhu, editor, Terzopoulos, Demetri, editor, Tygar, Dough, editor, Vardi, Moshe Y., editor, Weikum, Gerhard, editor, McGookin, David, editor, and Brewster, Stephen, editor

Published
2006
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37. Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

Author

Campbell, Craig, primary, McColl, Elaine, additional, McDermott, Michael P., additional, Martens, William B., additional, Guglieri, Michela, additional, Griggs, Robert C., additional, Straub, Volker, additional, Childs, Anne-Marie, additional, Ciafaloni, Emma, additional, Shieh, Perry B., additional, Spinty, Stefan, additional, Butterfield, Russell J., additional, Horrocks, Iain, additional, Roper, Helen, additional, Maggi, Lorenzo, additional, Baranello, Giovanni, additional, Flanigan, Kevin M., additional, Kuntz, Nancy L., additional, Manzur, Adnan Y., additional, Darras, Basil T., additional, Kang, Peter, additional, Mah, Jean K., additional, Mongini, Tiziana, additional, Ricci, Federica, additional, Morrison, Leslie, additional, Krzesniak-Swinarska, Monika, additional, von der Hagen, Maja, additional, Finkel, Richard S., additional, Kumar, Ashutosh, additional, Wicklund, Matthew, additional, McDonald, Craig M., additional, Henricson, Erik K., additional, Schara-Schmidt, Ulrike, additional, Wilichowski, Ekkehard, additional, Barohn, Richard J., additional, Statland, Jeffrey, additional, Kirschner, Janbernd, additional, Vita, Giuseppe, additional, Vita, Gian Luca, additional, Howard, James F., additional, Hughes, Imelda, additional, McMillan, Hugh J., additional, Pegoraro, Elena, additional, Bello, Luca, additional, Burnette, W. Bryan, additional, Thangarajh, Mathula, additional, and Chang, Taeun, additional

Published
2021
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40. Psychometric properties of the PEDI-CAT for children and youth with spinal muscular atrophy

Author

Fragala-Pinkham, Maria, primary, Pasternak, Amy, additional, McDermott, Michael P., additional, Mirek, Elizabeth, additional, Glanzman, Allan M., additional, Montes, Jacqueline, additional, Dunaway Young, Sally, additional, Salazar, Rachel, additional, Quigley, Janet, additional, Riley, Susan O., additional, Chiriboga, Claudia A., additional, Finkel, Richard S., additional, Tennekoon, Gihan, additional, Martens, William B., additional, De Vivo, Darryl C., additional, and Darras, Basil T., additional

Published
2021
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41. Applying adaptive recognition of the learner’s vowel space to English pronunciation training of native speakers of Japanese

Author

Roy, D, Fragulis, G, Cantu Campos, HA, Martens, William L, Wang, Rui, Roy, D, Fragulis, G, Cantu Campos, HA, Martens, William L, and Wang, Rui

Abstract

When native speakers of Japanese are taught English as a second language, there are difficulties with their training in pronunciation of American English vowels that can be ameliorated though adaptive recognition of the learner’s vowel space. This paper reports on the development of an online Computer-Assisted Language Learning (CALL) environment that provides Japanese learners with customized target utterances of 12 single-syllable words that are synthesized according to an adaptive recognition of the learner’s vowel space. These customized target utterances provide each learner with examples of each of 12 American English monophthongs in consonant-vowel-consonant (CVC) context in order to sound as if they had been uttered by the learners themselves. This adaptive process was incorporated into a successfully developed tool for Computer-Assisted Pronunciation Training (CAPT) which gave more appropriate pronunciation targets to each learner, rather than forcing the learners to attempt to match the formant frequencies of their own utterances to those of the target utterances as produced by a speaker exhibiting a different vowel space (i.e., a speaker with a different vocal tract length).

Published
2021

45. Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes

Author

Young, Sally Dunaway, Pasternak, Amy, Duong, Tina, McGrattan, Katlyn E., Stranberg, Sarah, Maczek, Elizabeth, Dias, Courtney, Tang, Whitney, Parker, Dana, Levine, Alexis, Rohan, Alyssa, Wolford, Connie, Martens, William, McDermott, Michael P., Darras, Basil T., and Day, John W.

Abstract

Novel Spinal Muscular Atrophy (SMA) treatments have demonstrated improvements on motor measures that are clearly distinct from the natural history of progressive decline. Comparable measures are needed to monitor bulbar function, which is affected in severe SMA. To assess bulbar function with patient-reported outcome measures (PROs) and determine their relationships with clinical characteristics. We recruited 47 non-ambulatory participants (mean (SD) age = 29.8 (13.7) years, range = 10.3–73.2) with SMA. PROs including Voice Handicap Index (VHI) and Eating Assessment Tool-10 (EAT-10) were collected alongside clinical characteristics and standardized motor assessments. Associations were assessed using Spearman correlation coefficients and group comparisons were performed using Wilcoxon rank sum tests. A majority of the 47 participants were SMA type 2 (70.2%), non-sitters (78.7%), 3 copies of SMN2(77.5%), and using respiratory support (66.0%). A majority (94%) reported voice issues primarily in 8/30 VHI questions. Problems included: difficulty understanding me in a noisy room (87.2%); difficult for people to hear me (74.5%); and people ask me to repeat when speaking face-to-face (72.3%). A majority (85.1%) reported swallowing issues primarily in 3/10 EAT-10 questions: swallowing pills (68.1%); food sticks to my throat (66.0%); and swallowing solids (61.7%). The two PROs were moderately associated (rs = 0.66). Weaker individuals with SMA experience bulbar problems including difficulties with voice and swallowing. Further refinement and assessment of functional bulbar scales will help determine their relevance and responsiveness to changes in SMA. Additional study is needed to quantify bulbar changes caused by SMA and their response to disease-modifying treatments.

Published
2023
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46. A quantitative measure of handgrip myotonia in non-dystrophic myotonia

Author

Statland, Jeffrey M., Bundy, Brian N., Wang, Yunxia, Trivedi, Jaya R., Raja Rayan, Dipa, Herbelin, Laura, Donlan, Merideth, McLin, Rhonda, Eichinger, Katy J., Findlater, Karen, Dewar, Liz, Pandya, Shree, Martens, William B., Venance, Shannon L., Matthews, Emma, Amato, Anthony A., Hanna, Michael G., Griggs, Robert C., and Barohn, Richard J.

Published
2012
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49. Mexiletine in Myotonic Dystrophy Type 1

Author

Heatwole, Chad, primary, Luebbe, Elizabeth, additional, Rosero, Spencer, additional, Eichinger, Katy, additional, Martens, William, additional, Hilbert, James, additional, Dekdebrun, Jeanne, additional, Dilek, Nuran, additional, Zizzi, Christine, additional, Johnson, Nicholas, additional, Puwanant, Araya, additional, Tawil, Rabi, additional, Schifitto, Giovanni, additional, Beck, Christopher A., additional, Richeson, J. Franklin, additional, Zareba, Wojciech, additional, Thornton, Charles, additional, McDermott, Michael P., additional, and Moxley, Richard, additional

Published
2020
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