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 2016

Challenges of the set-up process for academic led international studies of rare diseases

by McColl, Elaine, Bushby, Kate, Guglieri, Michela, Davis, Becky, Watson, Gillian, Griggs, Robert, Hart, Kim
Published 2015

Improving recognition of Duchenne muscular dystrophy: a retrospective case note review

by van Ruiten, Henriette J A, Straub, Volker, Bushby, Kate, Guglieri, Michela
Published 2014

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 2015

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 2015

Muscle-Derived Proteins as Serum Biomarkers for Monitoring Disease Progression in Three Forms of Muscular Dystrophy

by Burch, Peter M., Pogoryelova, Oksana, Goldstein, Richard, Bennett, Donald, Guglieri, Michela, Straub, Volker, Bushby, Kate, Lochmüller, Hanns, Morris, Carl
Published 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

Neurocognitive Profiles in Duchenne Muscular Dystrophy and Gene Mutation Site

by D’Angelo, Maria Grazia, Lorusso, Maria Luisa, Civati, Federica, Comi, Giacomo Pietro, Magri, Francesca, Del Bo, Roberto, Guglieri, Michela, Molteni, Massimo, Turconi, Anna Carla, Bresolin, Nereo
Published 2011

Dystromirs as Serum Biomarkers for Monitoring the Disease Severity in Duchenne Muscular Dystrophy

by Zaharieva, Irina T., Calissano, Mattia, Scoto, Mariacristina, Preston, Mark, Cirak, Sebahattin, Feng, Lucy, Collins, James, Kole, Ryszard, Guglieri, Michela, Straub, Volker, Bushby, Kate, Ferlini, Alessandra, Morgan, Jennifer E., Muntoni, Francesco
Published 2013

Design, set-up and utility of the UK facioscapulohumeral muscular dystrophy patient registry

by Evangelista, Teresinha, Wood, Libby, Fernandez-Torron, Roberto, Williams, Maggie, Smith, Debbie, Lunt, Peter, Hudson, Judith, Norwood, Fiona, Orrell, Richard, Willis, Tracey, Hilton-Jones, David, Rafferty, Karen, Guglieri, Michela, Lochmüller, Hanns
Published 2016

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-...

by Cirak, Sebahattin, Arechavala-Gomeza, Virginia, Guglieri, Michela, Feng, Lucy, Torelli, Silvia, Anthony, Karen, Abbs, Stephen, Garralda, Maria Elena, Bourke, John, Wells, Dominic J, Dickson, George, Wood, Matthew JA, Wilton, Steve D, Straub, Volker, Kole, Ryszard, Shrewsbury, Stephen B, Sewry, Caroline, Morgan, Jennifer E, Bushby, Kate, Muntoni, Francesco
Published 2011