Long-term efficacy and safety of viltolarsen in the treatment of Duchenne muscular dystrophy

被引：0
|
作者：
Clemens, P. [1 ]
Connolly, A. [2 ]
Harper, A. [3 ]
Mah, J. [4 ]
McDonald, C. [5 ]
Rao, V. [6 ]
Smith, E. [7 ]
Zaidman, C. [8 ]
Nakagawa, T. [9 ]
Hoffman, E. [10 ]
机构：
[1] Univ Pittsburgh, Pittsburgh, PA USA
[2] Nationwide Childrens Hosp, Columbus, OH USA
[3] Childrens Hosp Richmond, Richmond, VA USA
[4] Univ Calgary, Calgary, AB, Canada
[5] Univ Calif Davis, Sacramento, CA 95817 USA
[6] Lurie Childrens Hosp Chicago, Chicago, IL USA
[7] Duke Univ, Med Ctr, Durham, NC USA
[8] Washington Univ, St Louis, MO 63110 USA
[9] NS Pharma, Paramus, NJ USA
[10] SUNY Binghamton, Sch Pharm, Binghamton, NY USA
来源：
NEUROMUSCULAR DISORDERS | 2021年 / 31卷
关键词：
D O I：
10.1016/j.nmd.2021.07.165
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
EP.140
引用
收藏
页码：S91 / S91
页数：1
相关论文
共 50 条
  • [1] Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy
    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, 2022, 9 (04) : 493 - 501
  • [2] Results of a Long-Term Efficacy and Safety Study of Viltolarsen in the Treatment of Duchenne Muscular Dystrophy Patients
    Rao, V
    Clemens, P.
    Connolly, A.
    Harper, A.
    Mah, J.
    Smith, E.
    McDonald, C.
    Zaidman, C.
    Nakagawa, T.
    Hoffman, E.
    ANNALS OF NEUROLOGY, 2021, 90 : S92 - S92
  • [3] Safety and efficacy of viltolarsen in ambulatory and nonambulatory males with Duchenne muscular dystrophy
    Harper, Amy D.
    Topaloglu, Haluk
    Mercuri, Eugenio
    Suslov, Vasiliy
    Wu, Liwen
    Ayanoglu, Cigdem Y.
    Tansey, Michael
    Previtera, Michelle L.
    Crozier, Robert A.
    Magnus, Leslie
    Clemens, Paula R.
    SCIENTIFIC REPORTS, 2024, 14 (01):
  • [4] Duchenne-Muscular Dystrophy: Long-Term efficacy of Steroid Treatment
    Manych, Matthias
    FORTSCHRITTE DER NEUROLOGIE PSYCHIATRIE, 2018, 86 (05) : 264 - 264
  • [5] Evolving Role of Viltolarsen for Treatment of Duchenne Muscular Dystrophy
    Vincik, LeighAnn Y.
    Dautel, Alexandra D.
    Staples, Abigail A.
    Lauck, Lillian V.
    Armstrong, Catherine J.
    Howard, Jeffery T.
    Mcgregor, David
    Ahmadzadeh, Shahab
    Shekoohi, Sahar
    Kaye, Alan D.
    ADVANCES IN THERAPY, 2024, 41 (04) : 1338 - 1350
  • [6] Viltolarsen (Viltepso) for Duchenne Muscular Dystrophy
    不详
    MEDICAL LETTER ON DRUGS AND THERAPEUTICS, 2020, 62 (1609): : 167 - 167
  • [7] Letter to the Editor: In response to PR Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy
    Muntoni, Francesco
    Straub, Volker
    Servais, Laurent
    Mercuri, Eugenio
    JOURNAL OF NEUROMUSCULAR DISEASES, 2023, 10 (06) : 1151 - 1153
  • [8] Short-term and long-term safety profile of Givinostat in Duchenne muscular dystrophy
    Vucinic, D.
    Nascimento, A.
    Finkel, R.
    Brandsema, J.
    Finanger, E.
    Harper, A.
    Acsadi, G.
    Nevo, Y.
    Houwen-van Opstal, S.
    Blaschek, A.
    Coceani, N.
    Cazzaniga, S.
    Bettica, P.
    Muelas, N.
    NEUROMUSCULAR DISORDERS, 2024, 43
  • [9] PREDNISONE TREATMENT IN DUCHENNE MUSCULAR-DYSTROPHY - LONG-TERM BENEFIT
    DESILVA, S
    DRACHMAN, DB
    MELLITS, D
    KUNCL, RW
    ARCHIVES OF NEUROLOGY, 1987, 44 (08) : 818 - 822
  • [10] Long-term effects of glucocorticoid treatment in patients with Duchenne muscular dystrophy
    不详
    BRITISH JOURNAL OF HOSPITAL MEDICINE, 2018, 79 (03) : 129 - 129
12345