Results 91 to 100 of about 2,627 (188)

Miglustat Treatment May Reduce Cerebrospinal Fluid Levels of the Axonal Degeneration Marker Tau in Niemann–Pick Type C

, 2011
Introduction: Niemann-Pick disease type C (NPC) is a lysosomal storage disorder that leads to progressive neurodegeneration. The glucosylceramide synthase blocker miglustat is being used to treat NPC, but monitoring of disease progression and treatment ...
Henrik Zetterberg, Simona Bianconi, Blennow, Kaj,, Zetterberg, Henrik,, Fu, Rao, Mattsson, Niklas,, Kaj Blennow, Månsson, Jan-Eric,, Jan-Eric Månsson, Porter, Forbes D, Rao Fu, Forbes D. Porter, Niklas Mattsson, Nicole M. Yanjanin, Bianconi, Simona, Yanjanin, Nicole M   +15 more
core   +1 more source

Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.

, 2004
It has been shown that treatment with miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) improves key clinical features of type I Gaucher disease after 1 year of treatment. This study reports longer-term efficacy and safety data.
Platt, Frances, Cox, TM, M. Maas, J. M. F. G. Aerts, Platt, FM, R. A. Dwek, Hollak, C, F. M. Platt, Elstein, D., R. H. Lachmann, Hrebicek, M., Lachmann, R. H., Zimran, A, Aerts, J. M. F. G., Zimran, A., M. Hrebicek, C. Hollak, Elstein, D, Platt, F. M., Dwek, RA, S. van Weely, Maas, M, Hollak, C., Dwek, R. A., Butters, T. D., van Weely, S., Hrebicek, M, T. D. Butters, Butters, TD, Cox, T. M., Lachmann, RH, A. Zimran, D. Elstein, Aerts, JM, van Weely, S, Maas, M., T. M. Cox   +36 more
core   +1 more source

Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data

Journal of Comparative Effectiveness Research
Aim: Late-onset Pompe disease is characterized by progressive loss of muscular and respiratory function. Until recently, standard of care was enzyme replacement therapy (ERT) with alglucosidase alfa.
Simon Shohet, Noemi Hummel, Shuai Fu, Ian Keyzor, Alasdair MacCulloch, Neil Johnson, Jeff Castelli, Ilona Czarny-Ozga, Tahseen Mozaffar, Howard Thom   +9 more
doaj   +1 more source

Results from a 9-year Intensive Safety Surveillance Scheme (IS(3) ) in miglustat (Zavesca(®) )-treated patients

, 2015
Following approval in the EU in 2002 and the USA in 2003, an Intensive Safety Surveillance Scheme (IS(3) ) was initiated to educate prescribers on the appropriate use of miglustat for the treatment of type I Gaucher disease (GD1), and to actively solicit
Muller, Audrey, Alsop, Jonathan, Hughes, Derralynn, Brand, Monika, Bembi, Bruno, van Schaik, Ivo N.   +5 more
core   +1 more source

Longitudinal changes in cerebellar and subcortical volumes in adult-onset Niemann–Pick disease type C patients treated with miglustat

, 2022
Niemann–Pick disease type C (NPC) is a rare neurovisceral disorder resulting in impaired intracellular lipid trafficking. The only disease-modifying treatment available to date is miglustat, an iminosugar inhibiting the accumulation of lipid by-products ...
M Walterfang (13387071), E A Bowman (13478404), Larry Abel (13056207), P Desmond (9722717), M Fahey (13425285), D Velakoulis (10456577)   +5 more
core  

Parallel improvement of sodium and chloride transport defects by miglustat (n-butyldeoxynojyrimicin) in cystic fibrosis epithelial cells.

, 2008
(IF : 4,00)International audienceCystic fibrosis, an autosomal recessive disease frequently diagnosed in the Caucasian population, is characterized by deficient Cl- transport due to mutations in the cystic fibrosis transmembrane conductance regulator ...
de Jonge, Hugo, R., Noël, Sabrina, Wilke, Martina, Bot, Alice, G. M., Becq, Frédéric   +4 more
core   +1 more source

Identification of serum protein biomarkers in individuals with Niemann-Pick disease, type C1

Biomarker Research
Background Niemann-Pick disease, type C1 (NPC1), is a rare, fatal, neurodegenerative lysosomal disorder caused by pathological variants in NPC1. Defects in lysosomal cholesterol transport result in the accumulation of unesterified cholesterol within the ...
Khushboo Singhal, Matthew T. Menold, Niamh X. Cawley, Kiersten Campbell, Nicole Y. Farhat, Derek Alexander, Ryan K. Dale, Forbes D. Porter   +7 more
doaj   +1 more source

Miglustat ameliorates isoproterenol-induced cardiac fibrosis via targeting UGCG

Molecular Medicine
Background Cardiac fibrosis is significant global health problem, which is associated with numerous cardiovascular diseases, and ultimately leads to the progression to heart failure.
Jing Liu, Wenqi Li, Ran Jiao, Zhigang Liu, Tiantian Zhang, Dan Chai, Lingxin Meng, Zhongyi Yang, Yuming Liu, Hongliang Wu, Xiaoting Gu, Xiaohe Li, Cheng Yang   +12 more
doaj   +1 more source

MEMORY IMPAIRMENT IN NIEMANN-PICK C DISEASE: AMELIORATING EFFECTS OF MIGLUSTAT.

, 2009
Niemann–Pick Type C disease (NPC) is an autosomal recessive fatal disorder caused by mutations in the NPC1 gene. NPC shows abnormal intracellular accumulation of cholesterol and gangliosides associated with peripheral and central organ dysfunction. Among
Rufini, S, TANCREDI, VIRGINIA, RUFINI, STEFANO, Tancredi, V, D’Arcangelo G, D'ARCANGELO, GIOVANNA, De Chiara, G, Grossi, D, Frank,C, Merlo, D   +9 more
core   +1 more source

Long-term miglustat therapy in children with niemann-pick disease type C

, 2010
Long-term miglustat therapy in children with niemann-pick disease type
MC Patterson (14464527), R Giorgino (13479904), D Vecchio (13425981), H Chadha-Boreham (13479898), JE Wraith (14464524), Larry Abel (13056207), E Jacklin (13479895), C Luzy (13479901)   +7 more
core