Zobrazeno 1 - 10
of 43
pro vyhledávání: '"Mathula, Thangarajh"'
Association between neurodevelopmental impairments and motor function in Duchenne muscular dystrophy
Publikováno v:
Annals of Clinical and Translational Neurology, Vol 10, Iss 12, Pp 2285-2296 (2023)
Abstract Objective We explored various prognostic factors of motor outcomes in corticosteroid‐naive boys with Duchenne Muscular Dystrophy (DMD). Methods The associations between parent‐reported neurodevelopmental concerns (speech delay, speech an
Externí odkaz:
https://doaj.org/article/79829bd13c7b40b88426668627dcff0f
Autor:
Celeste Michelle Pilato, Melissa Sue Walker, Andrea M. Nguyen, McKay Elizabeth Hanna, Scott Lanxing Huang, Erika Morgan Lutins, M. Alex Meredith, Peter Jacob Haar, Mathula Thangarajh, Hope Theresa, Woon Nam Chow
Publikováno v:
Autopsy and Case Reports, Vol 10, Iss 4 (2021)
There is scant information about the comprehensive distribution of dystrophic muscles in muscular dystrophy. Despite different clinical presentations of muscular dystrophy, a recent multi-center study concluded that phenotypic distribution of dystrop
Externí odkaz:
https://doaj.org/article/17da2e2652c54fdf904ceaf73c497a66
Autor:
Mathula Thangarajh, Aaron J. Kaat, Genila Bibat, Jennifer Mansour, Katherine Summerton, Anthony Gioia, Carly Berger, Kristina K. Hardy, Kathryn R. Wagner
Publikováno v:
Annals of Clinical and Translational Neurology, Vol 6, Iss 9, Pp 1696-1706 (2019)
Abstract Objective We performed a prospective, cross‐sectional cognitive assessment in subjects with Duchenne Muscular Dystrophy (DMD) and their biological mothers. Methods Thirty subjects with out‐of‐frame mutations in the dystrophin (DMD) gen
Externí odkaz:
https://doaj.org/article/59905b583bb54600ac7fd367b5bea18f
Autor:
Amy Bartlett, Stephen J. Kolb, Allison Kingsley, Kathryn J. Swoboda, Sandra P. Reyna, Ai Sakonju, Basil T. Darras, Richard Shell, Nancy Kuntz, Diana Castro, Susan T. Iannaccone, Julie Parsons, Anne M. Connolly, Claudia A. Chiriboga, Craig McDonald, W. Bryan Burnette, Klaus Werner, Mathula Thangarajh, Perry B. Shieh, Erika Finanger, Christopher S. Coffey, Jon W. Yankey, Merit E. Cudkowicz, Michelle M. McGovern, D. Elizabeth McNeil, W. David Arnold, John T. Kissel
Publikováno v:
Contemporary Clinical Trials Communications, Vol 11, Iss , Pp 113-119 (2018)
Background/Aims: Recruitment and retention of research participants are challenging and critical components of successful clinical trials and natural history studies. Infants with spinal muscular atrophy (SMA) have been a particularly challenging pop
Externí odkaz:
https://doaj.org/article/c20105f5211a4c60a677082f4b5666d9
Autor:
Rebecca A. Crow, Kimberly A. Hart, Michael P. McDermott, Rabi Tawil, William B. Martens, Barbara E. Herr, Elaine McColl, Jennifer Wilkinson, Janbernd Kirschner, Wendy M. King, Michele Eagle, Mary W. Brown, Deborah Hirtz, Hanns Lochmuller, Volker Straub, Emma Ciafaloni, Perry B. Shieh, Stefan Spinty, Anne-Marie Childs, Adnan Y. Manzur, Lucia Morandi, Russell J. Butterfield, Iain Horrocks, Helen Roper, Kevin M. Flanigan, Nancy L. Kuntz, Jean K. Mah, Leslie Morrison, Basil T. Darras, Maja von der Hagen, Ulrike Schara, Ekkehard Wilichowski, Tiziana Mongini, Craig M. McDonald, Giuseppe Vita, Richard J. Barohn, Richard S. Finkel, Matthew Wicklund, Hugh J. McMillan, Imelda Hughes, Elena Pegoraro, W. Bryan Burnette, James F. Howard, Mathula Thangarajh, Craig Campbell, Robert C. Griggs, Kate Bushby, Michela Guglieri
Publikováno v:
Trials, Vol 19, Iss 1, Pp 1-9 (2018)
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. Ov
Externí odkaz:
https://doaj.org/article/59fc2ba5f36543fab1efe37a189c7b3f
Autor:
Mathula Thangarajh, Aiping Zhang, Kirandeep Gill, Habtom W Ressom, Zhenzhi Li, Rency S Varghese, Eric P Hoffman, Kanneboyina Nagaraju, Yetrib Hathout, Simina M Boca
Publikováno v:
PLoS ONE, Vol 14, Iss 7, p e0219507 (2019)
Urine is increasingly being considered as a source of biomarker development in Duchenne Muscular Dystrophy (DMD), a severe, life-limiting disorder that affects approximately 1 in 4500 boys. In this study, we considered the mdx mice-a murine model of
Externí odkaz:
https://doaj.org/article/6ee45656fc6044f29c566c57134a1a71
Autor:
John W. Day, Mar Tulinius, Alan Pestronk, Tina Duong, Tulio E. Bertorini, Alberto Dubrovsky, Nanette C. Joyce, Anne M. Connolly, Hanna Kolski, Lauren P. Morgenroth, Hoda Abdel-Hamid, Ksenija Gorni, Craig M. McDonald, Erik K Henricson, Erik Landfeldt, Yoram Nevo, Jose Carlo, Sherilyn W. Driscoll, Laura McAdam, S. Chidambaranathan, Paula R. Clemens, Avital Cnaan, Jean Teasley, W. Douglas Biggar, Joel Iff, Andrew J. Kornberg, Nancy L. Kuntz, E. Henricson, Jean K. Mah, Carolina Tesi-Rocha, Robert T. Leshner, Mathula Thangarajh, Richard D. Webster, V. Vishwanathan, Monique M. Ryan, John B. Bodensteiner, Timothy Lotze, Richard T. Abresch, Peter I. Karachunski
Publikováno v:
Value Health
OBJECTIVES: The objective of this study was to examine the psychometric properties of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL 4.0 GCS) in Duchenne muscular dystrophy (DMD), a rare, severely debilitating, and ultimately
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2dd36dd2d6151eb55eab84c122ed70a8
https://doi.org/10.1016/j.jval.2021.05.016
https://doi.org/10.1016/j.jval.2021.05.016
Publikováno v:
Muscle Nerve
INTRODUCTION/AIMS: There is considerable heterogenicity in clinical outcomes in Duchenne Muscular Dystrophy (DMD). The aim of current study was to assess whether dystrophin gene (DMD) pathogenic variant location influences upper extremity and lower e
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::69c776b99a38bf19962d80d63e91a080
https://doi.org/10.1002/mus.27371
https://doi.org/10.1002/mus.27371
Autor:
Jos G.M. Hendriksen, Mathula Thangarajh, Hermien E. Kan, Francesco Muntoni, Dr. Y. Aoki, Dr P. Collin, Dr M. Colvin, Dr N. Doorenweerd, Prof A. Ferlini, Dr A. Goyenvalle, Dr J.J. Hendriksen, Dr J. Hoskin, Dr H.E. Kan, Mr F. Lamy, Dr K. Maresh, Prof F. Muntoni, Dr E.H. Niks, Prof U. Schara, Prof D. Skuse, Prof V. Straub, Prof S. Takeda, Dr M. Thangarajh, Dr N. Truba, Prof S. Tyagarajan, Dr C. Vaillend, Dr M. van Putten, Prof J. Vissing, E. Vroom
Publikováno v:
Neuromuscular Disorders
Neuromuscular Disorders, Elsevier, 2020, 30 (9), pp.782-794. ⟨10.1016/j.nmd.2020.08.357⟩
Neuromuscular Disorders, 30(9), 782-794. PERGAMON-ELSEVIER SCIENCE LTD
Neuromuscular Disorders, Elsevier, 2020, 30 (9), pp.782-794. ⟨10.1016/j.nmd.2020.08.357⟩
Neuromuscular Disorders, 30(9), 782-794. PERGAMON-ELSEVIER SCIENCE LTD
International audience; The role of brain dystrophin in muscular dystrophy: Implications for clinical care and translational research, Hoofddorp, the Netherlands, November 29 th-December 1 st 2019
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::575f77fed85a047101751fd1582bb226
https://doi.org/10.1016/j.nmd.2020.08.357
https://doi.org/10.1016/j.nmd.2020.08.357
Autor:
Jos Hendriksen, Mathula Thangarajh, Treat-Nmd, Michael P. McDermott, Robert C. Griggs, William B. Martens, Kimberly A. Hart
Publikováno v:
Neurology. 93:e1597-e1604
ObjectiveWe performed a prospective, cross-sectional analysis of neurodevelopmental concerns and psychosocial adjustment in relation to DMD mutations in young steroid-naive boys with dystrophinopathy.MethodsWe evaluated 196 steroid-naive boys with dy
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ff4ce97851c6536e6ce3a3ec5a5dbf33
https://doi.org/10.1212/wnl.0000000000008363
https://doi.org/10.1212/wnl.0000000000008363