Results from a 36-Week Open-Label Study of Recombinant Human Growth Hormone and Testosterone in Facioscapulohumeral Muscular Dystrophy (FSHD)

被引:0
|
作者
Heatwole, Chad [1 ]
Luebbe, Elizabeth [1 ]
Hamel, Johanna [1 ]
Mongiovi, Philip [1 ]
Ciafaloni, Emma [1 ]
Dilek, Nuran [1 ]
Martens, William [1 ]
Weber, David [2 ]
Hani, Rashid [1 ]
Allen, Jamie [1 ]
Smith, Claire [1 ]
Howell, Samantha [1 ]
Rosero, Spencer [1 ]
Eichinger, Katy [1 ]
Baker, Linday [1 ]
Dekdebrun, Jeanne [1 ]
Hilbert, Jim [1 ]
Varma, Anika [1 ]
McDermott, Michael [1 ]
Thornton, Charles [1 ]
Moxley, Richard [1 ]
机构
[1] Univ Rochester Med Ctr, Rochester, NY USA
[2] Childrens Hosp Philadelphia, Philadelphia, PA USA
关键词
D O I
10.1212/WNL.0000000000202958
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
S48.009
引用
收藏
页数:5
相关论文
共 50 条
  • [1] Results from a National Cross-Sectional Study of Disease-Burden in Facioscapulohumeral Muscular Dystrophy (FSHD)
    Heatwole, Chad
    Bode, Rita
    Martens, William
    McDermott, Michael
    Moxley, Richard
    Quinn, Christine
    Tawil, Alrabi
    Rothrock, Nan
    Vickrey, Barbara
    Victorson, David
    Johnson, Nicholas
    NEUROLOGY, 2012, 78
  • [2] Reduction of direct and indirect costs in patients with AS receiving etanercept: results from an open-label 36-week extension of the ASCEND study in four European countries
    Moots, Robert J.
    Ostor, Andrew J. K.
    Loft, Anne G. R.
    Jarvinen, Pentti
    Larsson, Per
    Ekelund, Mats
    Reynolds, Alan
    Kielar, Danuta
    Lindkvist, Rose-Marie
    Qvitzau, Susanne
    RHEUMATOLOGY, 2012, 51 (02) : 393 - 396
  • [3] Interim results from AIR, an open-label, single arm, 36-week ph 2 trial of C21 in subjects with idiopathic pulmonary fibrosis
    Maher, T.
    Ganslandt, C.
    Batta, R.
    Tornling, G.
    Bengtsson, T.
    Pophale, H.
    Rosendahl, E.
    Maher, T.
    EUROPEAN RESPIRATORY JOURNAL, 2022, 60
  • [4] Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study
    Goemans, Nathalie M.
    Tulinius, Mar
    van den Hauwe, Marleen
    Kroksmark, Anna-Karin
    Buyse, Gunnar
    Wilson, Rosamund J.
    van Deutekom, Judith C.
    de Kimpe, Sjef J.
    Lourbakos, Afrodite
    Campion, Giles
    PLOS ONE, 2016, 11 (09):
  • [5] An open-label pilot study of losmapimod to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments in participants with facioscapulohumeral muscular dystrophy type 1
    Kools, Joost
    Voermans, Nicol
    Jiang, John G.
    Mitelman, Olga
    Mellion, Michelle L.
    Ramana, Vivekananda
    van Engelen, Baziel G. M.
    JOURNAL OF THE NEUROLOGICAL SCIENCES, 2024, 462
  • [6] Assessment of user-friendliness of the Norditropin FlexPro for pediatric patients treated with recombinant human growth hormone: results of an open-label user survey
    Adachi, Masanori
    JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM, 2013, 26 (11-12): : 1105 - 1110
  • [7] Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study
    Clemens, Paula R.
    Rao, Vamshi K.
    Connolly, Anne M.
    Harper, Amy D.
    Mah, Jean K.
    McDonald, Craig M.
    Smith, Edward C.
    Zaidman, Craig M.
    Nakagawa, Tomoyuki
    Hoffman, Eric P.
    JOURNAL OF NEUROMUSCULAR DISEASES, 2023, 10 (03) : 439 - 447
  • [8] Six-Month, Prospective, Longitudinal, Open-Label Caffeine and Dextromethorphan Phenotyping Study in Children with Growth Hormone Deficiency Receiving Recombinant Human Growth Hormone Replacement
    Kennedy, Mary Jayne
    Davis, Daniel A.
    Smith, Ned
    Gaedigk, Andrea
    Pearce, Robin E.
    Kearns, Gregory L.
    CLINICAL THERAPEUTICS, 2008, 30 (09) : 1687 - 1699
  • [9] EFFECTS OF RESMETIROM (MGL-3196) ON HEPATIC FAT, LIPIDS, LIVER ENZYMES AND MARKERS OF LIVER FIBROSIS IN AN OPEN-LABEL 36-WEEK EXTENSION STUDY IN NASH PATIENTS
    Harrison, Stephen A.
    Bashir, Mustafa
    Moussa, Sam
    Taub, Rebecca A.
    Frias, Juan Pablo
    Alkhouri, Naim
    HEPATOLOGY, 2019, 70 : 169A - 170A
  • [10] Testosterone and erectile function in hypogonadal men unresponsive to tadalafil: results from an open-label uncontrolled study
    Yassin, AA
    Saad, F
    Diede, HE
    ANDROLOGIA, 2006, 38 (02) : 61 - 68