scholarly journals Successful Noninvasive Ventilation and Enzyme Replacement Therapy in an Adult Patient with Morbus Hunter

2011 ◽  
pp. 77-82 ◽  
Author(s):  
M. Westhoff ◽  
P. Litterst
Keyword(s):  
Enzyme Replacement Therapy ◽  
Adult Patient ◽  
Replacement Therapy ◽  
Noninvasive Ventilation ◽  
Enzyme Replacement

scholarly journals Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report

2016 ◽  
Vol 10 (1) ◽  
Author(s):  
Gaetano Giuffrida ◽  
Rita Lombardo ◽  
Ernesto Di Francesco ◽  
Laura Parrinello ◽  
Francesco Di Raimondo ◽  
...  
Keyword(s):  
Case Report ◽  
Enzyme Replacement Therapy ◽  
Adult Patient ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Enzyme Replacement ◽  
Type 1 Gaucher Disease

scholarly journals Initiation of Enzyme Replacement Therapy for an Adult Patient with Asymptomatic Type 1 Gaucher's Disease.

2001 ◽  
Vol 40 (8) ◽  
pp. 716-721 ◽  
Author(s):  
Naoki TANAKA ◽  
Hiroshi SAITO ◽  
Toshiro ITO ◽  
Kayoko MOMOSE ◽  
Fumihiro ISHIDA ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Adult Patient ◽  
Replacement Therapy ◽  
Gaucher's Disease ◽  
Gaucher’S Disease ◽  
Enzyme Replacement

scholarly journals Large Mesenteric Gaucheroma Responds to Substrate Reduction Therapy: A New Management of Gaucheromas

2020 ◽  
Author(s):  
Shoji Yano ◽  
Kathryn Moseley ◽  
Neha Mahajan ◽  
Mikako Warren ◽  
Linda Vachon
Keyword(s):  
Combination Therapy ◽  
Enzyme Replacement Therapy ◽  
Lymph Nodes ◽  
Adult Patient ◽  
Replacement Therapy ◽  
Substrate Reduction Therapy ◽  
Protein Losing Enteropathy ◽  
Enzyme Replacement ◽  
Substrate Reduction ◽  
Type 3

AbstractGaucheromas, which are pseudotumors consisting of a cluster of Gaucher cells, are rare complications in Gaucher's disease (GD) and reported in patients treated with enzyme replacement therapy (ERT). Gaucheromas commonly develop in the lymph nodes in the mesenteric and mediastinal regions and can cause serious complications including protein-losing enteropathy. A large mesenteric Gaucheroma showed a significant reduction in size after initiation of substrate reduction therapy (SRT) with eliglustat in an adult patient with GD type 3. Combination therapy with ERT and SRT should be considered to prevent Gaucheromas in patients with GD.

Cardiac and motor improvement with enzyme replacement therapy in a patient with infantile-onset Pompe disease

2005 ◽  
Vol 36 (02) ◽  
Author(s):  
M Smitka ◽  
M von der Hagen ◽  
A Kaindl ◽  
C Gilitzer ◽  
J Dumontier ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Enzyme Replacement ◽  
Motor Improvement

RESPONSE TO ENZYME REPLACEMENT THERAPY IN PATIENTS WITH GAUCHER DISEASE TYPE I

2019 ◽  
Vol 22 (06) ◽  
pp. 103-117
Author(s):  
Mays Al-Tai ◽  
Deia Al-Asady ◽  
Rula Hamid
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Disease Type ◽  
Type I ◽  
Enzyme Replacement

Two siblings with attenuated MPS II form: Long term enzyme replacement therapy

2021 ◽  
Vol 132 (2) ◽  
pp. S107-S108
Author(s):  
Nato Vashakmadze ◽  
Leyla Namazova-Baranova ◽  
Natalia Zhurkova ◽  
Olga Gordeeva ◽  
Nina Fedorova ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Enzyme Replacement ◽  
Mps Ii
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