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Showing 25 - 36 of 85 results
Publication

Medical management of muscle weakness in Duchenne muscular dystrophy

Sarah R Rivera, Sumit K Jhamb, Hoda Z Abdel-Hamid, Gyula Acsadi, John Brandsema, Emma Ciafaloni, Basil T Darras, Susan T Iannaccone, Chamindra G Konersman, Nancy L Kuntz, Craig M McDonald, Julie A Parsons, Carolina Tesi Rocha, Craig M Zaidman, Russell J Butterfield, Anne M Connolly, Katherine D Mathews

Publication

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study

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

Publication

The CINRG Becker Natural History Study: Baseline characteristics

Paula R Clemens, Gabriela Niizawa, Jia Feng, Julaine Florence, Andrea Smith DʼAlessandro, Lauren P Morgenroth, Ksenija Gorni, Michela Guglieri, Anne Connolly, Matthew Wicklund, Tulio Bertorini, Jean K Mah, Mathula Thangarajh, Edward Smith, Nancy Kuntz, Craig M McDonald, Erik K Henricson, Saila Upadhyayula, Barry Byrne, Georgios Manousakis, Amy Harper, Elena Bravver, Susan Iannaccone, Christopher Spurney, Avital Cnaan, Heather Gordish-Dressman

Publication

Combination molecular therapies for type 1 spinal muscular atrophy.

Yohei Harada, Vamshi K Rao, Kapil Arya, Nancy L Kuntz, Christine J DiDonato, Galia Napchan-Pomerantz, Amit Agarwal, Vikki Stefans, Masahisa Katsuno, Aravindhan Veerapandiyan

Publication

Spinal muscular atrophy care in the COVID-19 pandemic era.

Aravindhan Veerapandiyan, Anne M Connolly, Richard S Finkel, Kapil Arya, Katherine D Mathews, Edward C Smith, Diana Castro, Russell J Butterfield, Julie A Parsons, Laurent Servais, Nancy Kuntz, Vamshi K Rao, John F Brandsema, Eugenio Mercuri, Emma Ciafaloni

Publication

Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis.

Robert J Graham, Francesco Muntoni, Imelda Hughes, Sabrina W Yum, Nancy L Kuntz, Michele L Yang, Barry J Byrne, Suyash Prasad, Rachel Alvarez, Casie A Genetti, Tmirah Haselkorn, Emma S James, Laurie B LaRusso, Mojtaba Noursalehi, Salvador Rico, Alan H Beggs

Publication

Utility and practice of electrodiagnostic testing in the pediatric population: An AANEM consensus statement

Peter B Kang, Hugh J McMillan, Nancy L Kuntz, Tanya J Lehky, Katharine E Alter, Kevin F Fitzpatrick, Charbel El Kosseifi, Susana Quijano-Roy

Publication

Pulsed glucocorticoids enhance dystrophic muscle performance through epigenetic-metabolic reprogramming

Mattia Quattrocelli, Aaron S Zelikovich, Zhen Jiang, Clara Bien Peek, Alexis R Demonbreun, Nancy L Kuntz, Grant D Barish, Saptarsi M Haldar, Joseph Bass, Elizabeth M McNally

Publication

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.

Eric P Hoffman, Benjamin D Schwartz, Laurel J Mengle-Gaw, Edward C Smith, Diana Castro, Jean K Mah, Craig M McDonald, Nancy L Kuntz, Richard S Finkel, Michela Guglieri, Katharine Bushby, Mar Tulinius, Yoram Nevo, Monique M Ryan, Richard Webster, Andrea L Smith, Lauren P Morgenroth, Adrienne Arrieta, Maya Shimony, Catherine Siener, Mark Jaros, Phil Shale, John M McCall, Kanneboyina Nagaraju, John van den Anker, Laurie S Conklin, Avital Cnaan, Heather Gordish-Dressman, Jesse M Damsker, Paula R Clemens

Publication

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.

Darryl C De Vivo, Enrico Bertini, Kathryn J Swoboda, Wuh-Liang Hwu, Thomas O Crawford, Richard S Finkel, Janbernd Kirschner, Nancy L Kuntz, Julie A Parsons, Monique M Ryan, Russell J Butterfield, Haluk Topaloglu, Tawfeg Ben-Omran, Valeria A Sansone, Yuh-Jyh Jong, Francy Shu, John F Staropoli, Douglas Kerr, Alfred W Sandrock, Christopher Stebbins, Marco Petrillo, Gabriel Braley, Kristina Johnson, Richard Foster, Sarah Gheuens, Ishir Bhan, Sandra P Reyna, Stephanie Fradette, Wildon Farwell

Publication

Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.

Anne M Connolly, Craig M Zaidman, Paul T Golumbek, Mary M Cradock, Kevin M Flanigan, Nancy L Kuntz, Richard S Finkel, Craig M McDonald, Susan T Iannaccone, Pallavi Anand, Catherine A Siener, Julaine M Florence, Linda P Lowes, Lindsay N Alfano, Linda B Johnson, Alina Nicorici, Leslie L Nelson, Jerry R Mendell

Publication

Moderate exercise improves function and increases adiponectin in the mdx mouse model of muscular dystrophy.

Aaron S Zelikovich, Mattia Quattrocelli, Isabella M Salamone, Nancy L Kuntz, Elizabeth M McNally