Results 101 to 110 of about 3,855 (201)

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

Enfermedad de Gaucher [PDF]

, 2015
La Enfermedad de Gaucher (EG) pertenece al grupo de las Enfermedades por Depósito Lisosomal (EDL), en las que existe un error en el metabolismo celular, de modo que hay déficit o se presenta un trastorno funcional de la enzima Glucocerebrosidasa.Como ...
Eusebio Ponce, Emiliana, García Frade, Luis Javier   +1 more
core   +1 more source

Growth in expenditure on high cost drugs in Australia [PDF]

The paper provides an overview of the expenditure of high cost drugs in Australia and examines the average annual growth of these programs. The outlook for expenditure on high cost medicines and possible policy responses is also considered.
Rebecca de Boer, Roy Harvey
core  

Pathogenic mycobacteria achieve cellular persistence by inhibiting the Niemann-Pick Type C disease cellular pathway [version 1; referees: 2 approved, 2 approved with reservations] [PDF]

, 2016
et al,, Ory, Daniel S
core   +1 more source

Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data. [PDF]

J Comp Eff Res
Fu S, Hummel N, Shohet S, Johnson N, MacCulloch A, Castelli J, Kerr W, Fox B, Gielen V.   +8 more
europepmc   +1 more source

Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials. [PDF]

Ther Adv Rare Dis
Andersen H, Díaz-Manera J, Goker-Alpan O, Mozaffar T, Sitaraman Das S, Fox B, Amon F, O'Brien-Prince K, Goldman M, Holdbrook F, Jain V, Byrne BJ.   +11 more
europepmc   +1 more source

Arimoclomol and levacetylleucine for the treatment of neurologic manifestations of Niemann-Pick disease type C: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG). [PDF]

Genet Med Open
Rodriguez-Gil JL, Smith WE, Chang IJ, Mendelsohn BA, ACMG Therapeutics Committee5∗documents@acmg.net.   +4 more
europepmc   +1 more source

Malaltia de Gaucher: avaluació del tractament [PDF]

, 2012
Almazán, Cari, Palacio, Núria, Sunyer, Berta   +2 more
core  

Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients [PDF]

core   +1 more source

Observational cohort study of the natural history of Niemann-Pick disease type C in the UK: a 5-year update from the UK clinical database [PDF]

, 2015
Jackie Imrie, Kate Strong, Lesley Heptinstall, Stephen Knight   +3 more
core   +1 more source