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Ibrutinib for the Management of Schnitzler Syndrome: A Novel Therapy for a Rare Condition von Jani, Prachi, Vissing, Megan B, Ahmed, Salman, Sluzevich, Jason C, Aulakh, Sonikpreet, Alegria, Victoria, Ailawadhi, Meghna, Chanan-Khan, Asher, Ailawadhi, Sikander

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Safety and efficacy of omaveloxolone in patients with mitochondrial myopathy: MOTOR trial von Madsen, Karen L., Buch, Astrid E., Cohen, Bruce H., Falk, Marni J., Goldsberry, Angela, Goldstein, Amy, Karaa, Amel, Koenig, Mary K., Muraresku, Colleen C., Meyer, Colin, O'Grady, Megan, Scaglia, Fernando, Shieh, Perry B., Vockley, Jerry, Zolkipli-Cunningham, Zarazuela, Haller, Ronald G., Vissing, John

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Aberrant regulation of epigenetic modifiers contributes to the pathogenesis in patients with selenoprotein N‐related myopathies von Bachmann, Christoph, Noreen, Faiza, Voermans, Nicol C., Schär, Primo L., Vissing, John, Fock, Johanna M., Bulk, Saskia, Kusters, Benno, Moore, Steven A., Beggs, Alan H., Mathews, Katherine D., Meyer, Megan, Genetti, Casie A., Meola, Giovanni, Cardani, Rosanna, Mathews, Emma, Jungbluth, Heinz, Muntoni, Francesco, Zorzato, Francesco, Treves, Susan

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Prospective observational study of FKRP-related limb-girdle muscular dystrophy R9: A GRASP consortium study von Alfano, Lindsay N, James, Meredith K, Grosfjeld Petersen, Kristine, Rudolf, Karen, Vissing, John, Augsburger, Renee, Mozaffar, Tahseen, Jones, Aileen, Butler, Amanda, Laubscher, Katie M, Mockler, Shelley R H, Mathews, Katherine D, Iammarino, Megan A, Reash, Natalie F, Pietruszewski, Lindsay, Lowes, Linda P, Strahler, Talia, Wicklund, Matthew, Hunn, Stephanie, Weihl, Conrad C, Sasidharan, Sandhya, Currence, Melissa, Statland, Jeffrey M, Stinson, Nikia, Holzer, Megan, Leung, Doris G, Lott, Donovan J, Kang, Peter B, Holsten, Scott, Desai, Urvi, Johnson, Nicholas E

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