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Showing 37 - 48 of 85 results
Publication

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.

Laurie S Conklin, Jesse M Damsker, Eric P Hoffman, William J Jusko, Panteleimon D Mavroudis, Benjamin D Schwartz, Laurel J Mengle-Gaw, Edward C Smith, Jean K Mah, Michela Guglieri, Yoram Nevo, Nancy Kuntz, Craig M McDonald, Mar Tulinius, Monique M Ryan, Richard Webster, Diana Castro, Richard S Finkel, Andrea L Smith, Lauren P Morgenroth, Adrienne Arrieta, Maya Shimony, Mark Jaros, Phil Shale, John M McCall, Yetrib Hathout, Kanneboyina Nagaraju, John van den Anker, Leanne M Ward, Alexandra Ahmet, Michaelyn R Cornish, Paula R Clemens

Publication

Novel methods of imaging and analysis for the thermoregulatory sweat test.

Michael S Carroll, David W Reed, Nancy L Kuntz, Debra E Weese-Mayer

Publication

Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!

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

Publication

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

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

Publication

A multicenter, retrospective medical record review of X‐linked myotubular myopathy: The recensus study

Alan H Beggs, Barry J Byrne, Sabine De Chastonay, Tmirah Haselkorn, Imelda Hughes, Emma S James, Nancy L Kuntz, Jennifer Simon, Lindsay C Swanson, Michele L Yang, Zi-Fan Yu, Sabrina W Yum, Suyash Prasad

Publication

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Eugenio Mercuri, Basil T Darras, Claudia A Chiriboga, John W Day, Craig Campbell, Anne M Connolly, Susan T Iannaccone, Janbernd Kirschner, Nancy L Kuntz, Kayoko Saito, Perry B Shieh, Már Tulinius, Elena S Mazzone, Jacqueline Montes, Kathie M Bishop, Qingqing Yang, Richard Foster, Sarah Gheuens, C Frank Bennett, Wildon Farwell, Eugene Schneider, Darryl C De Vivo, Richard S Finkel

Publication

Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening.

Jacqueline Glascock, Jacinda Sampson, Amanda Haidet-Phillips, Anne Connolly, Basil Darras, John Day, Richard Finkel, R Rodney Howell, Katherine Klinger, Nancy Kuntz, Thomas Prior, Perry B Shieh, Thomas O Crawford, Douglas Kerr, Jill Jarecki

Publication

Natural history of infantile-onset spinal muscular atrophy.

Stephen J Kolb, Christopher S Coffey, Jon W Yankey, Kristin Krosschell, W David Arnold, Seward B Rutkove, Kathryn J Swoboda, Sandra P Reyna, Ai Sakonju, Basil T Darras, Richard Shell, Nancy Kuntz, Diana Castro, Julie Parsons, Anne M Connolly, Claudia A Chiriboga, Craig McDonald, W Bryan Burnette, Klaus Werner, Mathula Thangarajh, Perry B Shieh, Erika Finanger, Merit E Cudkowicz, Michelle M McGovern, D Elizabeth McNeil, Richard Finkel, Susan T Iannaccone, Edward Kaye, Allison Kingsley, Samantha R Renusch, Vicki L McGovern, Xueqian Wang, Phillip G Zaworski, Thomas W Prior, Arthur H M Burghes, Amy Bartlett, John T Kissel

Publication

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Richard S Finkel, Eugenio Mercuri, Basil T Darras, Anne M Connolly, Nancy L Kuntz, Janbernd Kirschner, Claudia A Chiriboga, Kayoko Saito, Laurent Servais, Eduardo Tizzano, Haluk Topaloglu, Már Tulinius, Jacqueline Montes, Allan M Glanzman, Kathie Bishop, Z John Zhong, Sarah Gheuens, C Frank Bennett, Eugene Schneider, Wildon Farwell, Darryl C De Vivo

Publication

Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.

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

Publication

Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy

Luca Bello, Kevin M Flanigan, Robert B Weiss, Pietro Spitali, Annemieke Aartsma-Rus, Francesco Muntoni, Irina Zaharieva, Alessandra Ferlini, Eugenio Mercuri, Sylvie Tuffery-Giraud, Mireille Claustres, Volker Straub, Hanns Lochmüller, Andrea Barp, Sara Vianello, Elena Pegoraro, Jaya Punetha, Heather Gordish-Dressman, Mamta Giri, Craig M McDonald, Eric P Hoffman

Publication

International consensus guidance for management of myasthenia gravis: Executive summary

Donald B Sanders, Gil I Wolfe, Michael Benatar, Amelia Evoli, Nils E Gilhus, Isabel Illa, Nancy Kuntz, Janice M Massey, Arthur Melms, Hiroyuki Murai, Michael Nicolle, Jacqueline Palace, David P Richman, Jan Verschuuren, Pushpa Narayanaswami