Ritualized into adulthood: the scarcity of youth-focused rites of passage in America von Burrow, Heather Marie

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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind,... von Schoser, Benedikt, Roberts, Mark, Byrne, Barry J, Sitaraman, Sheela, Jiang, Hai, Laforêt, Pascal, Toscano, Antonio, Castelli, Jeff, Díaz-Manera, Jordi, Goldman, Mitchell, van der Ploeg, Ans T, Bratkovic, Drago, Kuchipudi, Srilakshmi, Mozaffar, Tahseen, Kishnani, Priya S, Sebok, Agnes, Pestronk, Alan, Dominovic-Kovacevic, Aleksandra, Khan, Aneal, Koritnik, Blaž, Tard, Celine, Lindberg, Christopher, Quinn, Colin, Eldridge, Crystal, Bodkin, Cynthia, Reyes-Leiva, David, Hughes, Derralynn, Stefanescu, Ela, SALORT-CAMPANA, Emmanuelle, Butler, Ernest, Bouhour, Francoise, Kim, Gee, Konstantinos Papadimas, George, Parenti, Giancarlo, Bartosik-Psujek, Halina, Kushlaf, Hani, Akihiro, Hashiguchi, Lau, Heather, Pedro, Helio, Andersen, Henning, Amartino, Hernan, Shiraishi, Hideaki, Kobayashi, Hiroshi, Tarnev, Ivaylo, Vengoechea, Jaime, Avelar, Jennifer, Shin, Jin-Hong, Cauci, Jonathan, Alonso-Pérez, Jorge, Janszky, Jozsef, Berthy, Julie, Cornelia, Kornblum, Gutschmidt, Kristina, Claeys, Kristl, Judit Molnar, Maria, Wencel, Marie, Tarnopolsky, Mark, Dimachkie, Mazen, Tchan, Michel, Freimer, Miriam, Longo, Nicola, Vidal-Fernandez, Nuria, Musumeci, Olimpia, Goker-Alpan, Ozlem, Deegan, Patrick, Clemens, Paula R., Roxburgh, Richard, Henderson, Robert, Hopkin, Robert, Sacconi, Sabrina, Fecarotta, Simona, Attarian, Shahram, Wenninger, Stephan, Dearmey, Stephanie, Hiwot, Tarekegn, Burrow, Thomas, Ruck, Tobias, Sawada, Tomo, Laszlo, Vescei, Löscher, Wolfgang, Chien, Yin-Hsiu

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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07) von Schoser, Benedikt, Kishnani, Priya S., Bratkovic, Drago, Byrne, Barry J., Claeys, Kristl G., Díaz-Manera, Jordi, Laforêt, Pascal, Roberts, Mark, Toscano, Antonio, van der Ploeg, Ans T., Castelli, Jeff, Goldman, Mitchell, Holdbrook, Fred, Sitaraman Das, Sheela, Wasfi, Yasmine, Mozaffar, Tahseen

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Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease von Kishnani, Priya S., Byrne, Barry J., Claeys, Kristl G., Díaz-Manera, Jordi, Dimachkie, Mazen M., Kushlaf, Hani, Mozaffar, Tahseen, Roberts, Mark, Schoser, Benedikt, Hummel, Noemi, Kopiec, Agnieszka, Holdbrook, Fred, Shohet, Simon, Toscano, Antonio

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Meaningful and Measurable Health Domains in Huntington’s Disease: Large-Scale Validation of the Huntington’s Disease Health-Related Quality of Life Questionnaire Across Severity St... von Ho, Aileen K., Landwehrmeyer, G. Bernhard, Rae, Daniela, Simpson, Sheila A., de Souza (Keylock), Jenny, Coulthard, Elizabeth, Gethin, Louise, Barker, Roger A., Fisher, Kate, Goodman, Anna, Hill, Susan, Swain, Rachel, Guzman, Natalie Valle, Callaghan, Jenny, Fullam, Ruth, Rosser, Anne, Tonner, Alison, Edwards, Maureen, Ho, Carrie, Pearson, Pauline, Foster, Jillian, McKenzie, Sue, Rothery, Jean, Deith, Catherine, Ireland, Jane, Harding, Alison, Laver, Fiona, Silva, Mark, Rowett, Liz, Musgrave, Hannah, Toscano, Jean, Gerrans, Emily, Anjum, Uruj, Coebergh, Jan, Eddy, Charlotte, Rose, Sarah, Andrews, Thomasin, Dougherty, Andrew, Golding, Charlotte, Lashwood, Alison, Ruddy, Deborah, Santhouse, Alastair, Whaite, Anna, Doherty, Karen, Haider, Salman, Robertson, Nicola, Tabrizi, Sarah, Taylor, Rachel, Bek, Judith, Craufurd, David, Johnson, Liz, Jones, Mary, Krishnamoorthy, Ashok, Oughton, Emma, Partington-Jones, Lucy, Westmoreland, Leann, Davidson, Lynn, Davison, Jill, Morgan, Karen, Savage, Lois, Wood, Josh, Siuda, Gill, Dixon, Kathryn, Armstrong, Richard, Burn, John, Weekes, Rebecca, Craven, Janet, Verstraelen (Ritchie), Nicola, Barrett, Wendy, Bandmann, Oliver, Fairtlough, Helen, Fillingham, Kay, Foustanos, Isabella, Gill, Paul, Kazoka, Mbombe, Peppa, Nadia, Quarrell, Oliver, Tidswell, Katherine, Gunner, Kerry, Harris, Kayla, Anderson, Mary, Keys, Liane, Smalley, Sarah, El-Nimr, George, Duffell, Allison, Kennedy (nee Smith), Karen, Bucher, Walter, de Schepper, Beatrice, Eden, John, Hendrikx, Victor, King, Diana, Lenon-Bird, Anne, Rapaille, Lilliane, Santini, Helen, Sasinkova, Pavla, Soltysiak, Beverley, Smith, Steve, van der Leer, Hans, van der Meer, Lucienne, Wooldridge, Michael

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