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SavePhase 1 Open-Label Study of Omigapil in Patients With LAMA2- or COL6-Related Dystrophy
Foley, A. Reghan; Yun, Pomi; Leach, Meganne E.; Neuhaus, Sarah B.; Averion, Gilberto V.; Hu, Ying; Hayes, Leslie H.; Donkervoort, Sandra; Jain, Minal S.; Waite, Melissa; Parks, Rebecca; Bharucha-Goebel, Diana X.; Mayer, Oscar H.; Zou, Yaqun; Fink, Margaret; DeCoster, Jameice; Mendoza, Christopher; Arevalo, Cynthia; Hausmann, Rudolf; Petraki, Diana; Cheung, Ken; Bonnemann, Carsten G. Share Save Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 studyCarrillo, Nuria; Malicdan, May C.; Leoyklang, Petcharat; Shrader, Joseph A.; Joe, Galen; Slota, Christina; Perreault, John; Heiss, John D.; Class, Bradley; Liu, Chia-Ying; Bradley, Kennan; Jodarski, Colleen; Ciccone, Carla; Driscoll, Claire; Parks, Rebecca; Van Wart, Scott; Bayman, Levent; Coffey, Christopher S.; Quintana, Melanie; Berry, Scott M.; Huizing, Marjan; Gahl, William A. Share Save Share Save Share Save Upper extremity outcome measures for collagen VI-related myopathy and LAMA2-related muscular dystrophyBendixen, Roxanna M.; Butrum, Jocelyn; Jain, Mina S.; Parks, Rebecca; Hodsdon, Bonnie; Nichols, Carmel; Hsia, Michelle; Nelson, Leslie; Keller, Katherine C.; McGuire, Michelle; Elliott, Jeffrey S.; Linton, Melody M.; Arveson, Irene C.; Tounkara, Fatou; Vasavada, Ruhi; Harnett, Elizabeth; Punjabi, Monal; Donkervoort, Sandra; Dastgir, Jahannaz; Leach, Meganne E.; Rutkowski, Anne; Waite, Melissa; Collins, James; Boennemann, Carsten G.; Meilleur, Katherine G. Share Save Share Save Share Save Share Save Share Save Share Save 
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