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Experimental treatment for Niemann-Pick disease type C1 appears safe, effective

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

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Intrathecal hydroxyproyl-beta-cyclodextrin appears to slow progression of cognitive and speech deficits. Hearing loss was a notable adverse event but was'manageable'in this devastating disease, say investigators.Medscape Medical News
Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Neurology & Neurosurgery News Source Type: news
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
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
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
More News: American Health | Brain | Clinical Trials | National Institutes of Health (NIH) | Neurology | Niemann-Pick Disease