Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic

por James, Meredith K., Rose, Kristy, Alfano, Lindsay N., Reash, Natalie F., Eagle, Michelle, Lowes, Linda P.
Publicado 2021

Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases

por Duong, Tina, Krosschell, Kristin J., James, Meredith K., Nelson, Leslie, Alfano, Lindsay N., Eichinger, Katy, Mazzone, Elena, Rose, Kristy, Lowes, Linda P., Mayhew, Anna, Florence, Julaine, King, Wendy, Senesac, Claudia R., Eagle, Michelle
Publicado 2021

Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies

por James, Meredith K, Alfano, Lindsay N, Muni-Lofra, Robert, Reash, Natalie F, Sodhi, Jassi, Iammarino, Megan A, Moat, Dionne, Shannon, Kianna, McCallum, Michelle, Richardson, Mark, Eagle, Michelle, Straub, Volker, Marini-Bettolo, Chiara, Lowes, Linda P, Mayhew, Anna G
Publicado 2022

Development of a standard of care for patients with valosin-containing protein associated multisystem proteinopathy

por Korb, Manisha, Peck, Allison, Alfano, Lindsay N., Berger, Kenneth I., James, Meredith K., Ghoshal, Nupur, Healzer, Elise, Henchcliffe, Claire, Khan, Shaida, Mammen, Pradeep P. A., Patel, Sujata, Pfeffer, Gerald, Ralston, Stuart H., Roy, Bhaskar, Seeley, William W., Swenson, Andrea, Mozaffar, Tahseen, Weihl, Conrad, Kimonis, Virginia
Publicado 2022

Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints

por Murphy, Alexander P., Morrow, Jasper, Dahlqvist, Julia R., Stojkovic, Tanya, Willis, Tracey A., Sinclair, Christopher D. J., Wastling, Stephen, Yousry, Tarek, Hanna, Michael S., James, Meredith K., Mayhew, Anna, Eagle, Michelle, Lee, Laurence E., Hogrel, Jean‐Yves, Carlier, Pierre G., Thornton, John S., Vissing, John, Hollingsworth, Kieren G., Straub, Volker
Publicado 2019

Longitudinal Analysis of Respiratory Function of Different Types of Limb Girdle Muscular Dystrophies Reveals Independent Trajectories

por Muni-Lofra, Robert, Juanola-Mayos, Eduard, Schiava, Marianela, Moat, Dionne, Elseed, Maha, Michel-Sodhi, Jassi, Harris, Elizabeth, McCallum, Michelle, Moore, Ursula, Richardson, Mark, Trainor, Christina, Wong, Karen, Malinova, Monika, Bolano-Diaz, Carla, Keogh, Michael John, Ghimenton, Elisabetta, Verdu-Diaz, Jose, Mayhew, Anna, Guglieri, Michela, Straub, Volker, James, Meredith K., Marini-Bettolo, Chiara, Diaz-Manera, Jordi
Publicado 2023

Three‐year quantitative magnetic resonance imaging and phosphorus magnetic resonance spectroscopy study in lower limb muscle in dysferlinopathy

por Reyngoudt, Harmen, Smith, Fiona E., Caldas de Almeida Araújo, Ericky, Wilson, Ian, Fernández‐Torrón, Roberto, James, Meredith K., Moore, Ursula R., Díaz‐Manera, Jordi, Marty, Benjamin, Azzabou, Noura, Gordish, Heather, Rufibach, Laura, Hodgson, Tim, Wallace, Dorothy, Ward, Louise, Boisserie, Jean‐Marc, Le Louër, Julien, Hilsden, Heather, Sutherland, Helen, Canal, Aurélie, Hogrel, Jean‐Yves, Jacobs, Marni, Stojkovic, Tanya, Bushby, Kate, Mayhew, Anna, Straub, Volker, Carlier, Pierre G., Blamire, Andrew M.
Publicado 2022

Cardiac and pulmonary findings in dysferlinopathy: A 3‐year, longitudinal study

por Moore, Ursula, Fernandez‐Torron, Roberto, Jacobs, Marni, Gordish‐Dressman, Heather, Diaz‐Manera, Jordi, James, Meredith K., Mayhew, Anna G., Harris, Elizabeth, Guglieri, Michela, Rufibach, Laura E., Feng, Jia, Blamire, Andrew M., Carlier, Pierre G., Spuler, Simone, Day, John W., Jones, Kristi J., Bharucha‐Goebel, Diana X., Salort‐Campana, Emmanuelle, Pestronk, Alan, Walter, Maggie C., Paradas, Carmen, Stojkovic, Tanya, Mori‐Yoshimura, Madoka, Bravver, Elena, Pegoraro, Elena, Lowes, Linda Pax, Mendell, Jerry R., Bushby, Kate, Bourke, John, Straub, Volker
Publicado 2022

Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study

por Moore, Ursula R, Jacobs, Marni, Fernandez-Torron, Roberto, Jang, Jiji, James, Meredith K, Mayhew, Anna, Rufibach, Laura, Mittal, Plavi, Eagle, Michelle, Cnaan, Avital, Carlier, Pierre G, Blamire, Andrew, Hilsden, Heather, Lochmüller, Hanns, Grieben, Ulrike, Spuler, Simone, Tesi Rocha, Carolina, Day, John W, Jones, Kristi J, Bharucha-Goebel, Diana X, Salort-Campana, Emmanuelle, Harms, Matthew, Pestronk, Alan, Krause, Sabine, Schreiber-Katz, Olivia, Walter, Maggie C, Paradas, Carmen, Hogrel, Jean-Yves, Stojkovic, Tanya, Takeda, Shin’ichi, Mori-Yoshimura, Madoka, Bravver, Elena, Sparks, Susan, Diaz-Manera, Jordi, Bello, Luca, Semplicini, Claudio, Pegoraro, Elena, Mendell, Jerry R, Bushby, Kate, Straub, Volker
Publicado 2018

Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach

por Mayhew, Anna G., James, Meredith K., Moore, Ursula, Sutherland, Helen, Jacobs, Marni, Feng, Jia, Lowes, Linda Pax, Alfano, Lindsay N., Muni Lofra, Robert, Rufibach, Laura E., Rose, Kristy, Duong, Tina, Bello, Luca, Pedrosa-Hernández, Irene, Holsten, Scott, Sakamoto, Chikako, Canal, Aurélie, Sánchez-Aguilera Práxedes, Nieves, Thiele, Simone, Siener, Catherine, Vandevelde, Bruno, DeWolf, Brittney, Maron, Elke, Gordish-Dressman, Heather, Hilsden, Heather, Guglieri, Michela, Hogrel, Jean-Yves, Blamire, Andrew M., Carlier, Pierre G., Spuler, Simone, Day, John W., Jones, Kristi J., Bharucha-Goebel, Diana X., Salort-Campana, Emmanuelle, Pestronk, Alan, Walter, Maggie C., Paradas, Carmen, Stojkovic, Tanya, Mori-Yoshimura, Madoka, Bravver, Elena, Díaz-Manera, Jordi, Pegoraro, Elena, Mendell, Jerry R., Straub, Volker
Publicado 2022

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial

por Guglieri, Michela, Clemens, Paula R., Perlman, Seth J., Smith, Edward C., Horrocks, Iain, Finkel, Richard S., Mah, Jean K., Deconinck, Nicolas, Goemans, Nathalie, Haberlova, Jana, Straub, Volker, Mengle-Gaw, Laurel J., Schwartz, Benjamin D., Harper, Amy D., Shieh, Perry B., De Waele, Liesbeth, Castro, Diana, Yang, Michelle L., Ryan, Monique M., McDonald, Craig M., Tulinius, Mar, Webster, Richard, McMillan, Hugh J., Kuntz, Nancy L., Rao, Vashmi K., Baranello, Giovanni, Spinty, Stefan, Childs, Anne-Marie, Sbrocchi, Annie M., Selby, Kathryn A., Monduy, Migvis, Nevo, Yoram, Vilchez-Padilla, Juan J., Nascimento-Osorio, Andres, Niks, Erik H., de Groot, Imelda J.M., Katsalouli, Marina, James, Meredith K., van den Anker, Johannes, Damsker, Jesse M., Ahmet, Alexandra, Ward, Leanne M., Jaros, Mark, Shale, Phil, Dang, Utkarsh J., Hoffman, Eric P.
Publicado 2022

Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials

por Diaz-Manera, Jordi, Fernandez-Torron, Roberto, LLauger, Jaume, James, Meredith K, Mayhew, Anna, Smith, Fiona E, Moore, Ursula R, Blamire, Andrew M, Carlier, Pierre G, Rufibach, Laura, Mittal, Plavi, Eagle, Michelle, Jacobs, Marni, Hodgson, Tim, Wallace, Dorothy, Ward, Louise, Smith, Mark, Stramare, Roberto, Rampado, Alessandro, Sato, Noriko, Tamaru, Takeshi, Harwick, Bruce, Rico Gala, Susana, Turk, Suna, Coppenrath, Eva M, Foster, Glenn, Bendahan, David, Le Fur, Yann, Fricke, Stanley T, Otero, Hansel, Foster, Sheryl L, Peduto, Anthony, Sawyer, Anne Marie, Hilsden, Heather, Lochmuller, Hanns, Grieben, Ulrike, Spuler, Simone, Tesi Rocha, Carolina, Day, John W, Jones, Kristi J, Bharucha-Goebel, Diana X, Salort-Campana, Emmanuelle, Harms, Matthew, Pestronk, Alan, Krause, Sabine, Schreiber-Katz, Olivia, Walter, Maggie C, Paradas, Carmen, Hogrel, Jean-Yves, Stojkovic, Tanya, Takeda, Shin’ichi, Mori-Yoshimura, Madoka, Bravver, Elena, Sparks, Susan, Bello, Luca, Semplicini, Claudio, Pegoraro, Elena, Mendell, Jerry R, Bushby, Kate, Straub, Volker
Publicado 2018

Assessment of disease progression in dysferlinopathy: A 1-year cohort study

por Moore, Ursula, Jacobs, Marni, James, Meredith K., Mayhew, Anna G., Fernandez-Torron, Roberto, Feng, Jia, Cnaan, Avital, Eagle, Michelle, Bettinson, Karen, Rufibach, Laura E., Lofra, Robert Muni, Blamire, Andrew M., Carlier, Pierre G., Mittal, Plavi, Lowes, Linda Pax, Alfano, Lindsay, Rose, Kristy, Duong, Tina, Berry, Katherine M., Montiel-Morillo, Elena, Pedrosa-Hernández, Irene, Holsten, Scott, Sanjak, Mohammed, Ashida, Ai, Sakamoto, Chikako, Tateishi, Takayuki, Yajima, Hiroyuki, Canal, Aurélie, Ollivier, Gwenn, Decostre, Valerie, Mendez, Juan Bosco, Sánchez-Aguilera Praxedes, Nieves, Thiele, Simone, Siener, Catherine, Shierbecker, Jeanine, Florence, Julaine M., Vandevelde, Bruno, DeWolf, Brittney, Hutchence, Meghan, Gee, Richard, Prügel, Juliana, Maron, Elke, Hilsden, Heather, Lochmüller, Hanns, Grieben, Ulrike, Spuler, Simone, Tesi Rocha, Carolina, Day, John W., Jones, Kristi J., Bharucha-Goebel, Diana X., Salort-Campana, Emmanuelle, Harms, Matthew, Pestronk, Alan, Krause, Sabine, Schreiber-Katz, Olivia, Walter, Maggie C., Paradas, Carmen, Hogrel, Jean-Yves, Stojkovic, Tanya, Takeda, Shin'ichi, Mori-Yoshimura, Madoka, Bravver, Elena, Sparks, Susan, Díaz-Manera, Jordi, Bello, Luca, Semplicini, Claudio, Pegoraro, Elena, Mendell, Jerry R., Bushby, Kate, Straub, Volker
Publicado 2019