Log in to search using one of your social media accounts:

 

New drug for Niemann-Pick disease type C1 appears safe, effective

Researchers report that an experimental drug appears to be safe and effective at slowing the progression of Niemann-Pick disease type C1.
Source: Health News - UPI.com - Category: Consumer Health News Source Type: news

Related Links:

ConclusionsThis is the largest screening study conducted to date in Turkey in the families of patients with NP-C with homozygous inheritance. We have reported heterozygote frequencies, identified a novel mutation, and detected new patients with NP-C. These findings will aid our understanding of NP-C and may lead to improved recognition and more timely diagnosis.
Source: Molecular Diagnosis and Therapy - Category: Molecular Biology Source Type: research
A new clinical trial conducted by Washington University School of Medicine in St Louis and the National Institutes of Health (NIH) in the US has shown that a drug called cyclodextrin slows progression of Niemann-Pick type C (NPC) disease.
Source: Drug Development Technology - Category: Pharmaceuticals Source Type: news
NIH-led clinical trial suggests that drug slows progression of rare neurological disease.
Source: National Institutes of Health (NIH) News Releases - Category: American Health Source Type: news
Publication date: Available online 10 August 2017 Source:The Lancet Author(s): Robert P Erickson, Maria Teresa Fiorenza
Source: The Lancet - Category: General Medicine Source Type: research
Publication date: Available online 10 August 2017 Source:The Lancet Author(s): Daniel S Ory, Elizabeth A Ottinger, Nicole Yanjanin Farhat, Kelly A King, Xuntian Jiang, Lisa Weissfeld, Elizabeth Berry-Kravis, Cristin D Davidson, Simona Bianconi, Lee Ann Keener, Ravichandran Rao, Ariane Soldatos, Rohini Sidhu, Kimberly A Walters, Xin Xu, Audrey Thurm, Beth Solomon, William J Pavan, Bernardus N Machielse, Mark Kao, Steven A Silber, John C McKew, Carmen C Brewer, Charles H Vite, Steven U Walkley, Christopher P Austin, Forbes D Porter Background Niemann-Pick disease, type C1 (NPC1) is a lysosomal storage disorder characterised...
Source: The Lancet - Category: General Medicine Source Type: research
Results of a small clinical trial in Washington State have shown promise to treat the neurogenerative condition called Niemann-Pick type C (NPC) that typically kills those afflicted before they turn 20.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
In conclusion, these findings demonstrate that NPC1 plays an important role in type I FCoV infection. U18666A or other cholesterol transport inhibitor may be considered as the antiviral drug for the treatment of cats with FIP. PMID: 28780424 [PubMed - as supplied by publisher]
Source: Antiviral Research - Category: Virology Authors: Tags: Antiviral Res Source Type: research
In conclusion, these findings demonstrate that NPC1 plays an important role in type I FCoV infection. U18666A or other cholesterol transport inhibitor may be considered as the antiviral drug for the treatment of cats with FIP.
Source: Antiviral Therapy - Category: Virology Source Type: research
In conclusion, these studies demonstrate that the Npc1 gene interacts with a HFD to promote weight gain through differential regulation of central energy metabolism pathways.
Source: AJP: Endocrinology and Metabolism - Category: Endocrinology Authors: Tags: Research Article Source Type: research
Publication date: Available online 1 August 2017 Source:Molecular Genetics and Metabolism Author(s): Shahzeb Hassan, Ellen Sidransky, Nahid Tayebi The study of the contribution of epigenetic mechanisms, including DNA methylation, histone modifications, and microRNAs, to human disease has enhanced our understanding of different cellular processes and diseased states, as well as the effect of environmental factors on phenotypic outcomes. Epigenetic studies may be particularly relevant in evaluating the clinical heterogeneity observed in monogenic disorders. The lysosomal storage disorders are Mendelian disorders characteriz...
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research
More News: Health | Niemann-Pick Disease