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

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

Autonomic Nervous System Dysregulation in Monozygous Twins With Nephropathic Cystinosis

Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids

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

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

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

Amy Bartlett, Ai Sakonju, Allison Kingsley, Anne M. Connolly, Basil T. Darras, Christopher S. Coffey, Claudia A. Chiriboga, Craig McDonald, D. Elizabeth McNeil, Diana Castro, Erika Finanger, John T. Kissel, Jon W. Yankey, Julie Parsons, Kathryn J. Swoboda, Klaus Werner, Mathula Thangarajh, Merit E. Cudkowicz, Michelle M. McGovern, Nancy Kuntz, Perry B. Shieh, Richard Shell, Sandra P. Reyna, Stephen J. Kolb, Susan T. Iannaccone, W. Bryan Burnette, W. David Arnold

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

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

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

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

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

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

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

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

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

Developing treatment guidelines for myasthenia gravis

Natural history of infantile-onset spinal muscular atrophy.

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