Zobrazeno 1 - 10
of 30
pro vyhledávání: '"Lauren P, Morgenroth"'
Autor:
Edward C Smith, Laurie S Conklin, Eric P Hoffman, Paula R Clemens, Jean K Mah, Richard S Finkel, Michela Guglieri, Mar Tulinius, Yoram Nevo, Monique M Ryan, Richard Webster, Diana Castro, Nancy L Kuntz, Laurie Kerchner, Lauren P Morgenroth, Adrienne Arrieta, Maya Shimony, Mark Jaros, Phil Shale, Heather Gordish-Dressman, Laura Hagerty, Utkarsh J Dang, Jesse M Damsker, Benjamin D Schwartz, Laurel J Mengle-Gaw, Craig M McDonald, CINRG VBP15 and DNHS Investigators
Publikováno v:
PLoS Medicine, Vol 17, Iss 9, p e1003222 (2020)
BackgroundTreatment with corticosteroids is recommended for Duchenne muscular dystrophy (DMD) patients to slow the progression of weakness. However, chronic corticosteroid treatment causes significant morbidities. Vamorolone is a first-in-class anti-
Externí odkaz:
https://doaj.org/article/b45ccb68e7754878b721301c83d6bdc1
Autor:
Craig M. McDonald, Eugenio Mercuri, Francesco Muntoni, Heather Gordish-Dressman, Lauren P. Morgenroth, Maria Bernadete Dutra de Resende, Shuizhen Zhou, Mathukumalli Lakshmi Neeharika, Kazuhiro Haginoya, Leigh Ramos-Platt, Paula Williams, Vinay Penematsa, Connie Chou, Min Lin, Shelley Johnson, Karyn Koladicz, Nicholas Mastrandrea, Christian Werner, Panayiota Trifillis
Publikováno v:
Wednesday, April 26.
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
Autor:
Simina M Boca, Maki Nishida, Michael Harris, Shruti Rao, Amrita K Cheema, Kirandeep Gill, Difei Wang, Lin An, Robinder Gauba, Haeri Seol, Lauren P Morgenroth, Erik Henricson, Craig McDonald, Jean K Mah, Paula R Clemens, Eric P Hoffman, Yetrib Hathout, Subha Madhavan
Publikováno v:
PLoS ONE, Vol 11, Iss 7, p e0159895 (2016)
[This corrects the article DOI: 10.1371/journal.pone.0153461.].
Externí odkaz:
https://doaj.org/article/4e0636dcbe404238b51da4164c431e3d
Autor:
Simina M Boca, Maki Nishida, Michael Harris, Shruti Rao, Amrita K Cheema, Kirandeep Gill, Haeri Seol, Lauren P Morgenroth, Erik Henricson, Craig McDonald, Jean K Mah, Paula R Clemens, Eric P Hoffman, Yetrib Hathout, Subha Madhavan
Publikováno v:
PLoS ONE, Vol 11, Iss 4, p e0153461 (2016)
Serum metabolite profiling in Duchenne muscular dystrophy (DMD) may enable discovery of valuable molecular markers for disease progression and treatment response. Serum samples from 51 DMD patients from a natural history study and 22 age-matched heal
Externí odkaz:
https://doaj.org/article/59c241aff29240059a2eadc8a6a161e9
Autor:
Matthew Wicklund, Jia Feng, Julaine Florence, Amy Harper, Nancy L. Kuntz, Anne M. Connolly, Saila Upadhyayula, Elena Bravver, Andrea Smith DʼAlessandro, Mathula Thangarajh, Tulio E. Bertorini, Craig M. McDonald, Ksenija Gorni, Jean K. Mah, Michela Guglieri, Edward C. Smith, Avital Cnaan, Paula R. Clemens, Lauren P. Morgenroth, Barry J. Byrne, Gabriela Niizawa, Erik K Henricson, Heather Gordish-Dressman, Susan T. Iannaccone, Christopher F. Spurney, Georgios Manousakis
Publikováno v:
Muscle Nerve
INTRODUCTION: We performed an observational, natural history study of males with in-frame dystrophin gene deletions causing Becker muscular dystrophy (BMD). METHODS: A prospective natural history study collected longitudinal medical, strength and tim
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::46c3761121f66b219824e10410ac7536
https://europepmc.org/articles/PMC7520226/
https://europepmc.org/articles/PMC7520226/
Autor:
Andrea Vianello, Francesca Magri, Luca Bello, Valeria A. Sansone, Tiziana Mongini, Eugenio Mercuri, Marina Pedemonte, S. Gandossini, Riccardo Masson, Marika Pane, Antonella Pini, Heather Gordish-Dressman, Sara Vianello, Adele D'Amico, Stefano C. Previtali, Aurora Fusto, Matteo Villa, Giacomo P. Comi, Claudio Bruno, Craig M. McDonald, Valentina Lanzillotta, Guja Astrea, Gian Luca Vita, Paola Tacchetti, Daniele Sabbatini, Luisa Politano, Enrico Bertini, Angela Berardinelli, Eric P. Hoffman, Andrea Barp, Lauren P. Morgenroth, Grazia D'Angelo, Beatrice Merlo, Federica Trucco, Sonia Messina, Elisa De Mattia, Emilio Albamonte, Fabrizio Rao, Giovanni Baranello, Elena Pegoraro
Publikováno v:
Annals of Clinical and Translational Neurology, Vol 7, Iss 5, Pp 786-798 (2020)
Annals of Clinical and Translational Neurology
Annals of Clinical and Translational Neurology
Objective: Respiratory insufficiency is a major complication of Duchenne muscular dystrophy (DMD).Its progression shows considerable interindividual variability, which has been less thoroughly characterized and understood than in skeletal muscle. We
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::91aab73f31387b267af70819a635b2b6
http://hdl.handle.net/11591/437225
http://hdl.handle.net/11591/437225
Autor:
Katy de Valle, Amy Harper, Cara L. Carty, Marni B. Jacobs, Mathula Thangarajh, K. Carroll, Jia Feng, Jean K. Mah, Paula R. Clemens, Anne M. Connolly, Mar Tulinius, Avital Cnaan, Yi-Wen Chen, Tina Duong, Monique M. Ryan, Lauren P. Morgenroth, Peter I. Karachunski, Edward C. Smith, Craig M. McDonald, Richard Webster, Michela Guglieri
Publikováno v:
Neurology. 90:e1333-e1338
ObjectivesTo investigate motor function associations with age, sex, and D4Z4 repeats among participants with early-onset facioscapulohumeral muscular dystrophy (FSHD) type 1 as defined by weakness onset before 10 years of age.MethodsWe collected stan
Autor:
Jean K. Mah, Heather Gordish-Dressman, Diana Castro, Nancy L. Kuntz, Utkarsh J. Dang, Laurie S. Conklin, Dnhs Investigators, Jesse M. Damsker, Monique M. Ryan, Paula R. Clemens, Maya Shimony, Phil Shale, Eric P. Hoffman, Mar Tulinius, Richard Webster, Laurel J Mengle-Gaw, Michela Guglieri, Mark Jaros, Laurie Kerchner, Richard S. Finkel, Antonio Arrieta, Edward C. Smith, Craig M. McDonald, Yoram Nevo, Benjamin D Schwartz, Laura Hagerty, Lauren P. Morgenroth
Publikováno v:
PLoS Medicine
PLoS Medicine, Vol 17, Iss 9, p e1003222 (2020)
PLoS Medicine, Vol 17, Iss 9, p e1003222 (2020)
Background Treatment with corticosteroids is recommended for Duchenne muscular dystrophy (DMD) patients to slow the progression of weakness. However, chronic corticosteroid treatment causes significant morbidities. Vamorolone is a first-in-class anti
Autor:
Paula R. Clemens, Craig M. Zaidman, Eric P. Hoffman, Jean K. Mah, Lauren P. Morgenroth, Amy Harper, Hironori Osaki, Youhei Satou, Edward C. Smith, Vamshi K. Rao, Craig M. McDonald, Anne M. Connolly, Taishi Yamashita
Publikováno v:
JAMA Neurol
Importance An unmet need remains for safe and efficacious treatments for Duchenne muscular dystrophy (DMD). To date, there are limited agents available that address the underlying cause of the disease. Objective To evaluate the safety, tolerability,