Gene therapy for type of blindness gets $850K price tag

Drugmaker says it lowered cost of treatment approved by the FDA last month, citing insurer concerns
Source: Health News: CBSNews.com - Category: Consumer Health News Source Type: news

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Publication date: March 2018Source: Progress in Retinal and Eye Research, Volume 63Author(s): Marina França Dias, Kwangsic Joo, Jessica A. Kemp, Silvia Ligório Fialho, Armando da Silva Cunha, Se Joon Woo, Young Jik KwonAbstractRetinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people worldwide. It is characterized with progressive loss of rods and cones and causes severe visual dysfunction and eventual blindness in bilateral eyes. In addition to more than 3000 genetic mutations from about 70 genes, a wide genetic overlap with other types of retinal dystrophies has been report...
Source: Progress in Retinal and Eye Research - Category: Opthalmology Source Type: research
Fight Aging! provides a weekly digest of news and commentary for thousands of subscribers interested in the latest longevity science: progress towards the medical control of aging in order to prevent age-related frailty, suffering, and disease, as well as improvements in the present understanding of what works and what doesn't work when it comes to extending healthy life. Expect to see summaries of recent advances in medical research, news from the scientific community, advocacy and fundraising initiatives to help speed work on the repair and reversal of aging, links to online resources, and much more. This content is...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
(Michigan State University) A Michigan State University veterinary ophthalmologist has modified a gene therapy that reverses blindness in dogs that have a certain form of a disease known as progressive retinal atrophy, or PRA, and is now looking to advance the treatment for human use. Simon Petersen-Jones in the College of Veterinary Medicine has received a five-year, $8.2 million grant from the National Institutes of Health to further the therapy for people who have a type of retinitis pigmentosa.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
The objective here is a set of tests that (a) match up to the expected outcome based on human trials of mitochondrially targeted antioxidants, and (b) that anyone can run without the need to involve a physician, as that always adds significant time and expense. These tests are focused on the cardiovascular system, particularly measures influenced by vascular stiffness, and some consideration given to parameters relevant to oxidative stress and the development of atherosclerosis. A standard blood test, with inflammatory markers. An oxidized LDL cholesterol assessment. Resting heart rate and blood pressure. Heart r...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Authors: Sahel JA, Léveillard T Abstract Retinal degenerative diseases are a major cause of untreatable blindness due to a loss of photoreceptors. Recent advances in genetics and gene therapy for inherited retinal dystrophies (IRDs) showed that therapeutic gene transfer holds a great promise for vision restoration in people with currently incurable blinding diseases. Due to the huge genetic heterogeneity of IRDs that represents a major obstacle for gene therapy development, alternative therapeutic approaches are needed. This review focuses on the rescue of cone function as a therapeutic option for maintainin...
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research
Many of the methods by which aging can be modestly slowed in laboratory species are characterized by increased cellular housekeeping: more repair, more clearance of broken molecular machinery, more removal of metabolic waste. The extended life span produced by calorie restriction appears to depend on this increase: it doesn't happen in mice in which housekeeping processes are disabled. Most of the work on cellular housekeeping in aging is focused on autophagy, responsible for removing protein aggregates and cellular structures. The proteasome is a part of a separate system of housekeeping that deals with broken or otherwis...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs
Spark's first product, Luxturna, was approved by the FDA in December to treat an inherited retinal disorder that leads to blindness if untreated. The first patients have been treated with the gene therapy, which is now available at a select group of medical centers.
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
This study demonstrates that small peptide domains derived from native protein amelogenin can be utilized to construct a mineral layer on damaged human enamel in vitro. Six groups were prepared to carry out remineralization on artificially created lesions on enamel: (1) no treatment, (2) Ca2+ and PO43- only, (3) 1100 ppm fluoride (F), (4) 20 000 ppm F, (5) 1100 ppm F and peptide, and (6) peptide alone. While the 1100 ppm F sample (indicative of common F content of toothpaste for homecare) did not deliver F to the thinly deposited mineral layer, high F test sample (indicative of clinical varnish treatment) formed mainly C...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Cell implants, gene therapy, even optogenetics are making headway in clinical trials to treat various forms of blindness.
Source: The Scientist - Category: Science Tags: News Analysis Source Type: news
Nine-year-old Creed Pettit of Florida became one of the first people to receive a new a newly-approved gene therapy for his blindness at the University of Miami's Bascom Eye Institute yesterday.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
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