Avascular necrosis in neuronopathic Gaucher despite high-dose enzyme replacement therapy

被引:0
|
作者
Bailey, Lauren A. [1 ]
Sullivan, Jennifer [1 ]
Kishnani, Priya S. [1 ]
机构
[1] Duke Univ, Med Ctr, Durham, NC USA
关键词
D O I
10.1016/j.ymgme.2014.12.019
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
17
引用
收藏
页码:S17 / S17
页数:1
相关论文
共 50 条
  • [1] Bone manifestations in neuronopathic Gaucher disease while receiving high-dose enzyme replacement therapy
    Potnis, Kunal C.
    Flueckinger, Lauren B.
    Ha, Christine, I
    Upadia, Jariya
    Frush, Donald P.
    Kishnani, Priya S.
    MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : 157 - 161
  • [2] Ethical considerations for enzyme replacement therapy in neuronopathic Gaucher disease
    Elstein, D
    Abrahamov, A
    Zimran, A
    CLINICAL GENETICS, 1998, 54 (03) : 179 - 184
  • [3] The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease
    Altarescu, G
    Hill, S
    Wiggs, E
    Jeffries, N
    Kreps, C
    Parker , CC
    Brady, RO
    Barton, NW
    Schiffmann, R
    Bannarjee, TK
    Crutchfield, K
    Frei, K
    McKee, MA
    Moore, DF
    Tournay, A
    JOURNAL OF PEDIATRICS, 2001, 138 (04): : 539 - 547
  • [4] Early initiation of high-dose oral ambroxol in combination with enzyme replacement therapy in a neuropathic Gaucher infant
    Chu, Shao-Yin
    Chien, Chun-Ching
    Hwu, Wuh-Liang
    Wang, Pen-Jung
    Chien, Yin-Hsiu
    BLOOD CELLS MOLECULES AND DISEASES, 2020, 81
  • [5] Dose and enzyme replacement therapy of M.Gaucher
    vom Dahl, S.
    Charrow, J.
    Hollak, C. E. M.
    Kacena, K.
    Mistry, P.
    Zhang, L.
    Zimran, A.
    Grabowski, G.
    MEDIZINISCHE KLINIK, 2009, 104 (01) : 95 - 95
  • [6] Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis
    Mistry, Pramod K.
    Deegan, Patrick
    Vellodi, Ashok
    Cole, J. Alexander
    Yeh, Michael
    Weinreb, Neal J.
    BRITISH JOURNAL OF HAEMATOLOGY, 2009, 147 (04) : 561 - 570
  • [7] High-dose enzyme replacement therapy in murine Hurler syndrome
    Ou, Li
    Herzog, Tyler
    Koniar, Brenda L.
    Gunther, Roland
    Whitley, Chester B.
    MOLECULAR GENETICS AND METABOLISM, 2014, 111 (02) : 116 - 122
  • [8] Gaucher disease: Enzyme therapy in the acute neuronopathic variant
    Prows, CA
    Sanchez, N
    Daugherty, C
    Grabowski, GA
    AMERICAN JOURNAL OF MEDICAL GENETICS, 1997, 71 (01): : 16 - 21
  • [9] IMPACT OF HIGH-DOSE INHALED CORTICOSTEROIDS: A CASE OF AVASCULAR NECROSIS
    Kim, J.
    Tsai, M.
    Nguyen, J.
    Jalali, J.
    Yusin, J.
    ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY, 2024, 133 (06) : S117 - S117
  • [10] High-Dose Ambroxol Therapy in Type 1 Gaucher Disease Focusing on Patients with Poor Response to Enzyme Replacement Therapy or Substrate Reduction Therapy
    Istaiti, Majdolen
    Frydman, Dafna
    Dinur, Tama
    Szer, Jeff
    Revel-Vilk, Shoshana
    Zimran, Ari
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2023, 24 (07)