Economic Evaluation in Duchenne Muscular Dystrophy: Model Frameworks for Cost-Effectiveness Analysis

by Landfeldt, Erik, Alfredsson, Lars, Straub, Volker, Lochmüller, Hanns, Bushby, Katharine, Lindgren, Peter
Published in Pharmacoeconomics (2016)

Quantifying the burden of caregiving in Duchenne muscular dystrophy

by Landfeldt, Erik, Lindgren, Peter, Bell, Christopher F., Guglieri, Michela, Straub, Volker, Lochmüller, Hanns, Bushby, Katharine
Published in J Neurol (2016)

Health‐related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross‐sectional study

by Landfeldt, Erik, Lindgren, Peter, Bell, Christopher F, Guglieri, Michela, Straub, Volker, Lochmüller, Hanns, Bushby, Katharine
Published in Dev Med Child Neurol (2015)

The burden of Duchenne muscular dystrophy: An international, cross-sectional study

by Landfeldt, Erik, Lindgren, Peter, Bell, Christopher F., Schmitt, Claude, Guglieri, Michela, Straub, Volker, Lochmüller, Hanns, Bushby, Katharine
Published 2014

Compliance to Care Guidelines for Duchenne Muscular Dystrophy

by Landfeldt, Erik, Lindgren, Peter, Bell, Christopher F., Schmitt, Claude, Guglieri, Michela, Straub, Volker, Lochmüller, Hanns, Bushby, Katharine
Published in J Neuromuscul Dis (2015)

Adult care for Duchenne muscular dystrophy in the UK

by Rodger, Sunil, Woods, Katherine L., Bladen, Catherine L., Stringer, Angela, Vry, Julia, Gramsch, Kathrin, Kirschner, Janbernd, Thompson, Rachel, Bushby, Katharine, Lochmüller, Hanns
Published in J Neurol (2014)

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management

by Birnkrant, David J, Bushby, Katharine, Bann, Carla M, Alman, Benjamin A, Apkon, Susan D, Blackwell, Angela, Case, Laura E, Cripe, Linda, Hadjiyannakis, Stasia, Olson, Aaron K, Sheehan, Daniel W, Bolen, Julie, Weber, David R, Ward, Leanne M
Published in Lancet Neurol (2018)

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

by Birnkrant, David J, Bushby, Katharine, Bann, Carla M, Apkon, Susan D, Blackwell, Angela, Brumbaugh, David, Case, Laura E, Clemens, Paula R, Hadjiyannakis, Stasia, Pandya, Shree, Street, Natalie, Tomezsko, Jean, Wagner, Kathryn R, Ward, Leanne M, Weber, David R
Published in Lancet Neurol (2018)

Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan

by Birnkrant, David J, Bushby, Katharine, Bann, Carla M, Apkon, Susan D, Blackwell, Angela, Colvin, Mary K, Cripe, Linda, Herron, Adrienne R, Kennedy, Annie, Kinnett, Kathi, Naprawa, James, Noritz, Garey, Poysky, James, Street, Natalie, Trout, Christina J, Weber, David R, Ward, Leanne M
Published in Lancet Neurol (2018)

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

by Hoffman, Eric P., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., Smith, Edward C., Castro, Diana, Mah, Jean K., McDonald, Craig M., Kuntz, Nancy L., Finkel, Richard S., Guglieri, Michela, Bushby, Katharine, Tulinius, Mar, Nevo, Yoram, Ryan, Monique M., Webster, Richard, Smith, Andrea L., Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Siener, Catherine, Jaros, Mark, Shale, Phil, McCall, John M., Nagaraju, Kanneboyina, van den Anker, John, Conklin, Laurie S., Cnaan, Avital, Gordish-Dressman, Heather, Damsker, Jesse M., Clemens, Paula R.
Published in Neurology (2019)

Ataluren treatment of patients with nonsense mutation dystrophinopathy

by Bushby, Katharine, Finkel, Richard, Wong, Brenda, Barohn, Richard, Campbell, Craig, Comi, Giacomo P, Connolly, Anne M, Day, John W, Flanigan, Kevin M, Goemans, Nathalie, Jones, Kristi J, Mercuri, Eugenio, Quinlivan, Ros, Renfroe, James B, Russman, Barry, Ryan, Monique M, Tulinius, Mar, Voit, Thomas, Moore, Steven A, Lee Sweeney, H, Abresch, Richard T, Coleman, Kim L, Eagle, Michelle, Florence, Julaine, Gappmaier, Eduard, Glanzman, Allan M, Henricson, Erik, Barth, Jay, Elfring, Gary L, Reha, Allen, Spiegel, Robert J, O'donnell, Michael W, Peltz, Stuart W, Mcdonald, Craig M, FOR THE PTC124-GD-007-DMD STUDY GROUP
Published in Muscle Nerve (2014)