Combination of high-dose ambroxol and ERT in Gaucher disease type 2: A nearly age-appropriate neurocognitive and motor development after three years of treatment

被引：0
|
作者：
Aries, Charlotte [1 ]
Lohmoeller, Benjamin [1 ]
Tiede, Stephan [2 ]
Taeuber, Karolin [3 ]
Rudolph, Cornelia [1 ]
Muschol, Nicole [1 ]
机构：
[1] Univ Med Ctr Hamburg Eppendorf, Dept Pediat, Int Ctr Lysosomal Disorders, Hamburg, Germany
[2] Univ Med Ctr Hamburg Eppendorf, Univ Childrens Res, Hamburg, Germany
[3] Univ Med Ctr Hamburg Eppendorf, Dept Child & Adolescent Psychiat & Psychotherapy, Hamburg, Germany
来源：
MOLECULAR GENETICS AND METABOLISM | 2022年 / 135卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2021.11.028
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
13
引用
收藏
页码：S19 / S19
页数：1
相关论文
共 4 条
  • [1] Successful ambroxol treatment in Gaucher disease type 2: Age-appropriate neurocognitive development after one year of follow-up
    Lohmoeller, Benjamin
    Ammer, Luise
    Aries, Charlotte
    Lindschau, Mona
    Stumpfe, Katharina
    Tiede, Stephan
    Muschol, Nicole
    MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S100 - S100
  • [2] Promising Effect of High Dose Ambroxol Treatment on Neurocognition and Motor Development in a Patient With Neuropathic Gaucher Disease 2
    Aries, Charlotte
    Lohmoeller, Benjamin
    Tiede, Stephan
    Taeuber, Karolin
    Hartmann, Guido
    Rudolph, Cornelia
    Muschol, Nicole
    FRONTIERS IN NEUROLOGY, 2022, 13
  • [3] Clinical and preclinical insights into high-dose ambroxol therapy for Gaucher disease type 2 and 3: A comprehensive systematic review
    den Hollander, Bibiche
    Le, Hoang Lan
    Swart, Eleonora L.
    Bikker, Hennie
    Hollak, Carla E. M.
    Brands, Marion M.
    MOLECULAR GENETICS AND METABOLISM, 2024, 143 (1-2)
  • [4] A Recent Update of Three Consecutive Prospective Trials with High-Dose Therapy and Autograft, without or with Rituximab, as Primary Treatment for Advanced-Stage Follicular Lymphoma (FL) Shows a Sizeable Group of Patients Surviving in Continuous Complete Remission up to 16 Years After the End of Treatment: Should We Still Consider FL An Incurable Disease ?
    Tarella, Corrado
    Ladetto, Marco
    Benedetti, Fabio
    Vitolo, Umberto
    Pulsoni, Alessandro
    Patti, Caterina
    Callea, Vincenzo
    Rambaldi, Alessandro
    Piccin, Andrea
    Magni, Michele
    Musso, Maurizio
    Iannitto, Emilio
    Spedini, Pierangelo
    Liberati, Anna Marina
    Ciceri, Fabio
    Gallamini, Andrea
    Rodeghiero, Francesco
    Gini, Guido
    De Crescenzo, Alberto
    Di Raimondo, Francesco
    Levis, Alessandro
    Chisesi, Teodoro
    Perrone, Tommasina
    Scalabrini, Delia Rota
    Rossi, Giuseppe
    Carella, Angelo Michele
    Parvis, Guido
    Majolino, Ignazio
    Passera, Roberto
    Ruella, Marco
    Pileri, Alessandro
    Gianni, Alessandro
    Corradini, Paolo
    BLOOD, 2009, 114 (22) : 363 - 364
1