Reply to "Levacetylleucine a game changer for Niemann-Pick disease type-C"

被引:0
作者
Matsuo, Muneaki [1 ]
Ishitsuka, Yoichi [2 ]
Irie, Tetsumi [2 ,3 ]
机构
[1] Saga Univ, Fac Med, Dept Pediat, 5-1-1 Nabeshima, Saga 8498501, Japan
[2] Kumamoto Univ, Grad Sch Pharmaceut Sci, Dept Clin Chem & Informat, Kumamoto, Japan
[3] Kumamoto Univ, Fac Life Sci, Dept Pharmaceut Packaging Technol, Kumamoto, Japan
关键词
D O I
10.1016/j.braindev.2024.104301
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
引用
收藏
页数:1
相关论文
共 50 条
  • [21] FELINE SPHINGOLIPIDOSIS RESEMBLING NIEMANN-PICK DISEASE TYPE-C
    LOWENTHAL, AC
    CUMMINGS, JF
    WENGER, DA
    THRALL, MA
    WOOD, PA
    DELAHUNTA, A
    [J]. ACTA NEUROPATHOLOGICA, 1990, 81 (02) : 189 - 197
  • [22] NEUROLOGICAL MANIFESTATIONS OF NIEMANN-PICK DISEASE TYPE-C IN CATS
    MUNANA, KR
    LUTTGEN, PJ
    THRALL, MA
    MITCHELL, TW
    WENGER, DA
    [J]. JOURNAL OF VETERINARY INTERNAL MEDICINE, 1994, 8 (02) : 117 - 121
  • [23] SPHINGOMYELINASE DEFECT IN NIEMANN-PICK DISEASE, TYPE-C, FIBROBLASTS
    BESLEY, GTN
    [J]. FEBS LETTERS, 1977, 80 (01) : 71 - 74
  • [24] Levacetylleucine (N-acetyl-L-leucine) for Niemann-Pick disease type C
    van Gool, Raquel
    Al-Hertani, Walla
    Bodamer, Olaf
    Upadhyay, Jaymin
    [J]. TRENDS IN PHARMACOLOGICAL SCIENCES, 2025, 46 (04) : 386 - 387
  • [25] Inflammation in the Niemann-Pick type-C brain
    Peake, Kyle Bradley
    Hayashi, Hideki
    Campenot, Robert
    Vance, Dennis
    Vance, Jean
    [J]. FASEB JOURNAL, 2007, 21 (05) : A234 - A235
  • [26] Niemann-Pick; Type C - Reply
    Shulman, LM
    David, NJ
    Weiner, WJ
    [J]. NEUROLOGY, 1996, 46 (06) : 1786 - 1786
  • [27] FETAL ASCITES - AN UNUSUAL PRESENTATION OF NIEMANN-PICK DISEASE TYPE-C
    MACONOCHIE, IK
    CHONG, S
    MIELIVERGANI, G
    LAKE, BD
    MOWAT, AP
    [J]. ARCHIVES OF DISEASE IN CHILDHOOD, 1989, 64 (10) : 1391 - 1393
  • [28] NIEMANN-PICK TYPE-C DISEASE IS ASSOCIATED WITH NEUROFIBRILLARY TANGLE FORMATION
    AUER, I
    CURRY, B
    [J]. LABORATORY INVESTIGATION, 1995, 72 (01) : A134 - A134
  • [29] A DEFECT IN CHOLESTEROL ESTERIFICATION IN NIEMANN-PICK DISEASE (TYPE-C) PATIENTS
    PENTCHEV, PG
    COMLY, ME
    KRUTH, HS
    VANIER, MT
    WENGER, DA
    PATEL, S
    BRADY, RO
    [J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1985, 82 (23) : 8247 - 8251
  • [30] NIEMANN-PICK DISEASE (CROCKERS TYPE-C) - ULTRASTRUCTURAL STUDY OF A CASE
    PELLISSIER, JF
    HASSOUN, J
    GAMBARELLI, D
    BRYON, PA
    CASANOVA, P
    TOGA, M
    [J]. ACTA NEUROPATHOLOGICA, 1975, 34 (01) : 65 - 76