Niemann-Pick disease treatment: a systematic review of clinical trials. [revisión]
Por: Santos Lozano, Alejandro [Instituto de Investigación i+12]
| Sanchis Gomar, Fabián [Instituto de Investigación imas12] | Fiuza Luces, Carmen [Instituto de Investigación i+12] | Pareja Galeano, Helios [Instituto de Investigación imas12] | Garatachea, Nuria [Instituto de Investigación i+12] | Lucía, Alejandro [Instituto de Investigación i+12].
Colaborador(es): Instituto de Investigación imas12.
Tipo de material: 
ArtículoEditor: Annals of translational medicine, 2015Descripción: 3(22):360.Recursos en línea: Acceso libre Resumen: The aim of this systematic review was to analyse all the published clinical trials assessing treatments for Niemann-Pick (NP) disease. At present there are only trials investigating the treatment of NP disease type C. Furthermore, there is no uniformity among studies in treatment outcomes or in data analysis and presentation of results. Miglustat is able to delay neurodegeneration, with greater benefits in patients with a late onset of the disease and β-cyclodextrin-hydroxypropyl (HBP-CD) can attenuate clinical symptoms. As for cholesterol-lowering drugs, the combination of lovastatin, cholestyramine and nicotinic acid is the most effective one for lowering cholesterolemia. Further research is much needed, and ongoing trials using enzyme replacement therapy might hopefully show promising results in the foreseeable future.
| Tipo de ítem | Ubicación actual | Signatura | Estado | Fecha de vencimiento |
|---|---|---|---|---|
Revisión
|
PC17178 (Navegar estantería) | Disponible |
Formato Vancouver:
Santos Lozano A, Villamandos García D, Sanchís Gomar F, Fiuza Luces C, Pareja Galeano H, Garatachea N et al. Niemann-Pick disease treatment: a systematic review of clinical trials. Ann Transl Med. 2015 Dec;3(22):360.
PMID: 26807415
PMC4701532
Contiene 35 referencias
The aim of this systematic review was to analyse all the published clinical trials assessing treatments for Niemann-Pick (NP) disease. At present there are only trials investigating the treatment of NP disease type C. Furthermore, there is no uniformity among studies in treatment outcomes or in data analysis and presentation of results. Miglustat is able to delay neurodegeneration, with greater benefits in patients with a late onset of the disease and β-cyclodextrin-hydroxypropyl (HBP-CD) can attenuate clinical symptoms. As for cholesterol-lowering drugs, the combination of lovastatin, cholestyramine and nicotinic acid is the most effective one for lowering cholesterolemia. Further research is much needed, and ongoing trials using enzyme replacement therapy might hopefully show promising results in the foreseeable future.
No hay comentarios para este ejemplar.