Piezo1: opening the way to preventing muscle atrophy by Ravi Jagasia, Kathryn R. Wagner

Get full text

Racial and ethnic diversity in global neuroscience clinical trials by Loes Rutten-Jacobs, Tammy McIver, Adriana Reyes, Marta Pereira, Rachel Rosenthal, Christine T. Parusel, Kathryn R. Wagner, Rachelle Doody

Get full text

Novel FHL1 Mutation Associated With Hypertrophic Cardiomyopathy, Sudden Cardiac Death, and Myopathy by M. Scott Binder, MD, Emily Brown, MGC, CGC, Thomas Aversano, MD, Kathryn R. Wagner, MD, PhD, Hugh Calkins, MD, Andreas S. Barth, MD, PhD

Get full text

Meeting report: the 2020 FSHD International Research Congress by Michael Kyba, Robert J. Bloch, Julie Dumonceaux, Scott Q. Harper, Silvère M. van der Maarel, Francis M. Sverdrup, Kathryn R. Wagner, Baziel van Engelen, Yi-Wen Chen

Get full text

Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy by Sarah P. Sherlock, Jeffrey Palmer, Kathryn R. Wagner, Hoda Z. Abdel-Hamid, Cuixia Tian, Jean K. Mah, Francesco Muntoni, Michela Guglieri, Russell J. Butterfield, Lawrence Charnas, Shannon Marraffino

Get full text

Longitudinal functional and imaging outcome measures in FKRP limb-girdle muscular dystrophy by Doris G. Leung, Alex E. Bocchieri, Shivani Ahlawat, Michael A. Jacobs, Vishwa S. Parekh, Vladimir Braverman, Katherine Summerton, Jennifer Mansour, Genila Bibat, Carl Morris, Shannon Marraffino, Kathryn R. Wagner

Get full text

Engineering Skeletal Muscle Grafts with PAX7::GFP-Sorted Human Pluripotent Stem Cell-Derived Myogenic Progenitors on Fibrin Microfiber Bundles for Tissue Regeneration by Sarah M. Somers, Jordana Gilbert-Honick, In Young Choi, Emily K. W. Lo, HoTae Lim, Shaquielle Dias, Kathryn R. Wagner, Hai-Quan Mao, Patrick Cahan, Gabsang Lee, Warren L. Grayson

Get full text

Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial. by Francesco Muntoni, Michela Guglieri, Jean K Mah, Kathryn R Wagner, John F Brandsema, Russell J Butterfield, Craig M McDonald, Anna G Mayhew, Jeffrey P Palmer, Shannon Marraffino, Lawrence Charnas, Eugenio Mercuri

Get full text

Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial by Francesco Muntoni, Michela Guglieri, Jean K. Mah, Kathryn R. Wagner, John F. Brandsema, Russell J. Butterfield, Craig M. McDonald, Anna G. Mayhew, Jeffrey P. Palmer, Shannon Marraffino, Lawrence Charnas, Eugenio Mercuri

Get full text

Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT) by Raffaella Willmann, Joanne Lee, Cathy Turner, Kanneboyina Nagaraju, Annemieke Aartsma-Rus, Dominic J. Wells, Kathryn R. Wagner, Cristina Csimma, Volker Straub, Miranda D. Grounds, Annamaria De Luca, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

Get full text

Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study by Claudia A. Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S. Scalco, Kathryn R. Wagner, Francesco Muntoni, the JEWELFISH Study Group

Get full text

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study by Claudia A. Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S. Scalco, Kathryn R. Wagner, Francesco Muntoni, the JEWELFISH Study Group

Get full text

Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model by In Young Choi, HoTae Lim, Kenneth Estrellas, Jyothi Mula, Tatiana V. Cohen, Yuanfan Zhang, Christopher J. Donnelly, Jean-Philippe Richard, Yong Jun Kim, Hyesoo Kim, Yasuhiro Kazuki, Mitsuo Oshimura, Hongmei Lisa Li, Akitsu Hotta, Jeffrey Rothstein, Nicholas Maragakis, Kathryn R. Wagner, Gabsang Lee

Get full text

The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy by Francesco Muntoni, Barry J. Byrne, Hugh J. McMillan, Monique M. Ryan, Brenda L. Wong, Juergen Dukart, Amita Bansal, Valerie Cosson, Roxana Dreghici, Maitea Guridi, Michael Rabbia, Hannah Staunton, Giridhar S. Tirucherai, Karl Yen, Xiling Yuan, Kathryn R. Wagner, the Taldefgrobep Alfa Study Group

Get full text

iMyoblasts for ex vivo and in vivo investigations of human myogenesis and disease modeling by Dongsheng Guo, Katelyn Daman, Jennifer JC Chen, Meng-Jiao Shi, Jing Yan, Zdenka Matijasevic, Amanda M Rickard, Monica H Bennett, Alex Kiselyov, Haowen Zhou, Anne G Bang, Kathryn R Wagner, René Maehr, Oliver D King, Lawrence J Hayward, Charles P Emerson Jr

Get full text