Pulmonary Function in Advanced Duchenne Muscular Dystrophy: Eteplirsen-Treated Patients versus Multiple Natural History Cohorts

被引:0
|
作者
Gordish-Dressman, Heather [1 ]
Henricson, Erik [2 ]
Han, Lixin [3 ]
Dugar, Ashish [3 ]
McDonald, Craig M. [2 ]
机构
[1] George Washington Univ, Sch Med & Hlth Sci, Washington, DC 20052 USA
[2] Univ Calif Davis, Davis, CA 95616 USA
[3] Sarepta Therapeut Inc, Cambridge, MA USA
[4] CINRG DNHS, Pittsburgh, PA USA
关键词
D O I
暂无
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
M279
引用
收藏
页码:S219 / S220
页数:2
相关论文
共 50 条
  • [21] Correlation of Bone Mineral Density with Pulmonary Function in Advanced Duchenne Muscular Dystrophy
    Lee, Jang Woo
    Cho, Han Eol
    Kang, Seong-Woong
    Choi, Won Ah
    Suh, Mi Ri
    Kim, Bitnarae
    PM&R, 2021, 13 (02) : 166 - 170
  • [22] Natural history of respiratory function changes in patients with Duchenne muscular dystrophy not using glucocorticoid steroids
    Buyse, G.
    Voit, T.
    Schara, U.
    Straathof, C.
    D'Angelo, M.
    Bernert, G.
    Cuisset, J.
    Finkel, R.
    Goemans, N.
    McDonald, C.
    Rummey, C.
    Meier, T.
    NEUROMUSCULAR DISORDERS, 2015, 25 : S305 - S305
  • [23] Effect of pulmonary function on right heart function in Duchenne muscular dystrophy patients
    Sharath Subramanian
    Kan N Hor
    Wojciech Mazur
    Suzanne Smart
    Tam Tran
    Nancy Halnon
    Michael D Taylor
    Linda Cripe
    Subha V Raman
    Journal of Cardiovascular Magnetic Resonance, 17 (Suppl 1)
  • [24] Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG Natural History Study: a matched cohort analysis
    Tulinius, M.
    Buccella, F.
    Desguerre, I.
    Kirschner, J.
    Mercuri, E.
    Muntoni, F.
    Osorio, A. Nascimento
    Morgenroth, L.
    Gordish-Dressman, H.
    Johnson, S.
    Werner, C.
    Jiang, J.
    Li, J.
    Jia, J.
    Trifillis, P.
    McDonald, C.
    NEUROMUSCULAR DISORDERS, 2022, 32 : S52 - S52
  • [25] Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG Natural History Study: a matched cohort analysis
    Tulinius, M.
    Buccella, F.
    Desguerre, I.
    Kirschner, J.
    Mercuri, E.
    Muntoni, F.
    Nascimento Osorio, A.
    Johnson, S.
    Werner, C.
    Kristensen, A.
    Jiang, J.
    Li, J.
    Trifillis, P.
    Santos, C.
    McDonald, C.
    NEUROMUSCULAR DISORDERS, 2021, 31 : S92 - S93
  • [26] Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE registry and the CINRG natural history study: a matched cohort analysis
    Tulinius, M.
    Buccella, F.
    Desguerre, I.
    Kirschner, J.
    Mercuri, E.
    Muntoni, F.
    Osorio, A. Nascimento
    Delage, A.
    Zhu, J.
    Kristensen, A.
    Trifillis, P.
    Santos, C.
    McDonald, C.
    NEUROMUSCULAR DISORDERS, 2019, 29 : S195 - S195
  • [27] Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE registry and CINRG natural history study: a matched cohort analysis
    Tulinius, M.
    Buccella, F.
    Desguerre, I.
    Kirschner, J.
    Mercuri, E.
    Muntoni, F.
    Osorio, A. Nascimento
    de Resende, M.
    Morgenroth, L.
    Gordish-Dressman, H.
    Johnson, S.
    Werner, C.
    Anbu, B.
    Liu, E.
    Rajbhandari, R.
    Trifillis, P.
    McDonald, C.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S143 - S143
  • [28] Pulmonary Function in Patients with Duchenne Muscular Dystrophy from the STRIDE Registry and CINRG Natural History Study: A Matched Cohort Analysis
    Tulinius, M.
    Buccella, F.
    Desguerre, I
    Kirschner, J.
    Mercuri, E.
    Muntoni, F.
    Osorio, Nascimento A.
    Johnson, S.
    Werner, C.
    Kristensen, A.
    Jiang, J.
    Li, J.
    Trifillis, P.
    Santos, C.
    McDonald, C.
    ANNALS OF NEUROLOGY, 2021, 90 : S154 - S154
  • [29] Pulmonary function is stable through week 120 in patients with Duchenne muscular dystrophy (DMD) treated with exon-skipping drug eteplirsen in phase 2b study
    Mendell, J. R.
    Lowes, L. P.
    Alfano, L.
    Saoud, J.
    Duda, P.
    Kaye, E.
    NEUROMUSCULAR DISORDERS, 2014, 24 (9-10) : 828 - 829
  • [30] Histologic muscular history in steroid-treated and untreated patients with Duchenne dystrophy
    Peverelli, Lorenzo
    Testolin, Silvia
    Villa, Luisa
    D'Amico, Adele
    Petrini, Stefania
    Favero, Chiara
    Magri, Francesca
    Morandi, Lucia
    Mora, Marina
    Mongini, Tiziana
    Bertini, Enrico
    Sciacco, Monica
    Comi, Giacomo P.
    Moggio, Maurizio
    NEUROLOGY, 2015, 85 (21) : 1886 - 1893