PROGRESSION OF DISEASE BURDEN AMONG BOYS WITH DUCHENNE MUSCULAR DYSTROPHY (DMD) ACCORDING TO AMBULATORY STATUS AND AGE AT LOSS OF AMBULATION (LoA)

被引：0
|
作者：
Gordish-Dressman, H. [1 ]
Zhang, R. [2 ]
Kamstra, R. [3 ]
Werner, C. [4 ]
Liu, E. [5 ]
Morrison, K. [3 ]
Tomazos, I [5 ]
机构：
[1] Childrens Natl Hosp, Washington, DC USA
[2] PTC Therapeut Sweden AB, Stockholm, AB, Sweden
[3] Precis Analyt Inc, Montreal, PQ, Canada
[4] PTC Therapeut, Frankfurt, Germany
[5] PTC Therapeut Inc, South Plainfield, NJ USA
来源：
VALUE IN HEALTH | 2024年 / 27卷 / 06期
关键词：
D O I：
暂无
中图分类号：
F [经济];
学科分类号：
02 ;
摘要：
CO120
引用
收藏
页码：S39 / S39
页数：1
相关论文
共 50 条
  • [41] EFFECT OF ATALUREN ON AGE AT LOSS OF AMBULATION IN NONSENSE MUTATION DUCHENNE MUSCULAR DYSTROPHY: OBSERVATIONAL DATA FROM THE STRIDE REGISTRY
    Delage, Abdallah
    Buccella, Filippo
    Desguerre, Isabelle
    Muntoni, Francesco
    Nascimento, Andres
    Tulinius, Mar
    Colucci, Salvatore
    Kristensen, Allan
    Able, Rich
    Santos, Claudio
    Trifillis, Panayiota
    Zhang, Olivia
    Mercuri, Eugenio
    MUSCLE & NERVE, 2019, 60 : S5 - S5
  • [42] Effect of Ataluren on Age at Loss of Ambulation in Nonsense Mutation Duchenne Muscular Dystrophy: Observational Data from the STRIDE Registry
    Delage, Abdallah
    Bucella, Filippo
    Desguerre, Isabelle
    Muntoni, Francesco
    Osorio, Andres Nascimento
    Tulinius, Mar
    Colucci, Salvatore
    Kristensen, Allan
    Santos, Claudio
    Schilling, Traci
    Trifillis, Panayiota
    Zhang, Olivia
    Mercuri, Eugenio
    NEUROLOGY, 2019, 92 (15)
  • [43] Age at First Symptoms/Signs and Loss of Ambulation in Duchenne-Becker Muscular Dystrophy: Data from the MD STARNet
    Ciafaloni, Emma
    McDermott, Michael
    Kumar, Anil
    Liu, Ke
    Pandya, Shree
    Westfield, Christine
    Fox, Deborah
    Caspers, Krisitn
    Puzhankara, Soman
    Cunniff, Christopher
    Mathews, Katherine
    West, Nancy
    Romitti, Paul
    NEUROLOGY, 2013, 80
  • [44] Non-ambulatory patients with Duchenne muscular dystrophy Recommendations for monitoring disease progression and course of treatment
    Flotats-Bastardas, Marina
    Ebrahimi-Fakhari, Daniel
    Bernert, Guenther
    Ziegler, Andreas
    Schlachter, Kurt
    Poryo, Martin
    Hahn, Andreas
    Meyer, Sascha
    NERVENARZT, 2019, 90 (08): : 817 - 823
  • [45] Age at First Symptoms/Signs and Loss of Ambulation in Duchenne-Becker Muscular Dystrophy: Data from the MD STARNet
    Ciafaloni, Emma
    McDermott, Michael
    Kumar, Anil
    Liu, Ke
    Pandya, Shree
    Westfield, Christine
    Fox, Deborah
    Caspers, Krisitn
    Puzhankara, Soman
    Cunniff, Christopher
    Mathews, Katherine
    West, Nancy
    Romitti, Paul
    NEUROLOGY, 2013, 80
  • [46] CLINICAL TRIAL READINESS IN NON-AMBULATORY BOYS AND MEN WITH DUCHENNE MUSCULAR DYSTROPHY: MDA-DMD NETWORK FOLLOW-UP
    Connolly, Anne M.
    Florence, Julaine M.
    Zaidman, Craig M.
    Golumbek, Paul T.
    Mendell, Jerry R.
    Flanigan, Kevin M.
    Karachunski, Peter I.
    Day, John W.
    Mcdonald, Craig M.
    Darras, Basil T.
    Kang, Peter B.
    Siener, Catherine A.
    Gadeken, Rebecca K.
    Anand, Pallavi
    Schierbecker, Jeanine R.
    Malkus, Elizabeth C.
    Lowes, Linda P.
    Alfano, Lindsay N.
    Johnson, Linda
    Nicorici, Alina
    Kelecic, Jason M.
    Quigley, Janet
    Pasternak, Amy E.
    Miller, J. Philip
    MUSCLE & NERVE, 2016, 54 (04) : 681 - 689
  • [47] Centiles by age for the North Star ambulatory assessment and the associated timed items in glucocorticoid treated boys with Duchenne muscular dystrophy
    Stimpson, G.
    Ridout, D.
    Wolfe, A.
    Milev, E.
    O'Reilly, E.
    Manzur, A.
    Cole, T.
    Muntoni, F.
    Baranello, G.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S83 - S83
  • [48] Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy: Data from the MD STARnet
    Ciafaloni, Emma
    Kumar, Anil
    Liu, Ke
    Pandya, Shree
    Westfield, Christina
    Fox, Deborah J.
    Conway, Kristin M. Caspers
    Cunniff, Christopher
    Mathews, Katherine
    West, Nancy
    Romitti, Paul A.
    McDermott, Michael P.
    JOURNAL OF PEDIATRIC REHABILITATION MEDICINE, 2016, 9 (01) : 5 - 11
  • [49] PEDIATRIC OUTCOMES DATA COLLECTION INSTRUMENT (PODCI) ASSESSMENT OF AMBULATORY BOYS TREATED WITH FORDADISTROGENE MOVAPARVOVEC (PF-06939926) FOR DUCHENNE MUSCULAR DYSTROPHY (DMD)
    Dorling, P.
    Ryan, K.
    Posner, N. W.
    Merla, V
    Mahn, M.
    Yong, F. H.
    VALUE IN HEALTH, 2022, 25 (12) : S395 - S395
  • [50] Progression to Loss of Ambulation Among Patients with Autosomal Recessive Limb-girdle Muscular Dystrophy: A Systematic Review
    Audhya, Ivana F.
    Cheung, Antoinette
    Szabo, Shelagh M.
    Flint, Emma
    Weihl, Conrad C.
    Gooch, Katherine L.
    JOURNAL OF NEUROMUSCULAR DISEASES, 2022, 9 (04) : 477 - 492
12345