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Spark's planned price for Luxturna gene therapy for rare blindness too high: ICER

(Reuters) - The proposed price for Spark Therapeutics Inc's groundbreaking one-time gene therapy for a rare form of blindness is far too high, an independent U.S. nonprofit organization that evaluates clinical and cost effectiveness of new medicines said on Friday.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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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
Photo courtesy of Mass. Eye &Ear Massachusetts Eye and Ear Infirmary made history this week, becoming the first institution to use an FDA-approved gene therapy to treat a patient with an inherited disease. Spark Therapeutics‘ (NSDQ:ONCE) Luxturna gene therapy, which was approved by the FDA in December, is designed to improve vision in patients with inherited retinal disease caused by a particular genetic mutation. Yesterday, Dr. Jason Comander injected the treatment into the eye of a 13-year-old boy from New Jersey. Get the full story at our sister site, Drug Delivery Business News. The post Mass. Eye &Ear ma...
Source: Mass Device - Category: Medical Devices Authors: Tags: Optical/Ophthalmic Pharmaceuticals Wall Street Beat Massachusetts Eye & Ear Infirmary sparktherapeutics Source Type: news
Philadelphia-based biotech Spark Therapeutics made history in December by winning FDA approval for the first gene therapy for an inherited disease. Now, Massachusetts Eye and Ear says it has performed the first post-approval injection of the drug, which treats a form of blindness and costs $850,000. The hospital announced on Tuesday that Jason Comander, the associate director of its inherited retinal disorders service, had performed the procedure on a 13-year-old boy from New Jersey. He is the first…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
(Children's Hospital Los Angeles) On March 20, 2018, surgeons at Children's Hospital Los Angeles, treated the hospital's first patient with gene therapy. The complex eye surgery was performed by dedicated retina specialist, Aaron Nagiel, MD, PhD , of The Vision Center at CHLA, on an adult patient with Leber's Congenital Amaurosis, a form of childhood blindness that affects the retina.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
(Massachusetts Eye and Ear Infirmary) Massachusetts Eye and Ear made medical history on Tuesday by performing the first post-FDA approval gene therapy for patients with a form of inherited blindness. The occasion marks the beginning of a new era in medicine, as it is the first time any FDA-approved gene therapy has been given to a patient for any inherited disease.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
In this study, we did not observe significant age-dependent upregulation of the prominent SASP cytokine Il6 in any tissue, although an upward trend was observed that was consistent in magnitude with previous observations in the heart and kidney. This modest age-related upward trend could be explained by a previous report which demonstrated that senescent cell-secreted IL-6 acts in an autocrine manner, reinforcing the senescent state, rather than inducing senescence or promoting dysfunction in neighboring cells. The decreased expression of Il6 with age we observed in the hypothalamus could be indicative of a lack or ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Set in its new home of the Francis Crick Institute, WIRED Health 2018 brought together world leaders and change-makers in cancer, aging, artificial intelligence, government, medical devices, and pharmaceuticals, to name but a few. Alongside the ...
Source: Medgadget - Category: Medical Devices Authors: Tags: Exclusive Source Type: blogs
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