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Gene Therapy for Blindness Appears Initially Effective, Says U.S. FDA

The treatment will be reviewed by an outside panel this week -- Read more on ScientificAmerican.com
Source: Scientific American - Category: Science Authors: Tags: Health Biotech Mind The Sciences Source Type: research

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GenSight Biologics, a biopharmaceutical company based in France, is developing gene therapies for rare diseases that cause blindness. The company has recently received approval in the UK for its Phase I/II PIONEER trial, which will test a treatment c...
Source: Medgadget - Category: Medical Devices Authors: Tags: Exclusive Genetics Ophthalmology Source Type: blogs
Authors: Zeitz C, Friedburg C, Preising MN, Lorenz B Abstract Congenital stationary night blindness (CSNB) is a clinically and genetically heterogeneous group of non-progressive retinal disorder with largely normal fundus appearance. The mode of inheritance can be autosomal dominant (adCSNB), autosomal recessive (arCSNB) or X-chromosomal (XLCSNB). Additional ocular signs can be myopia, hyperopia, strabismus, nystagmus and reduced visual acuity. The Riggs and Schubert-Bornschein form of CSNB can be discriminated by electroretinography. While the Riggs form represents a dysfunction of the rods, a signal transmission ...
Source: Klinische Monatsblatter fur Augenheilkunde - Category: Opthalmology Tags: Klin Monbl Augenheilkd Source Type: research
(Reuters) - Spark Therapeutics Inc, whose breakthrough gene therapy to treat a rare form of blindness was approved by U.S. regulators in December, has licensed rights to the drug in markets outside the United States to Switzerland-based Novartis AG, it said on Wednesday.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Source: FT.com - Drugs and Healthcare - Category: Pharmaceuticals Source Type: news
(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
(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
SAN FRANCISCO (Reuters) - Global drugmakers are looking to a tiny biotech's $850,000 therapy for a rare type of blindness as a model for getting paid for highly expensive – and effective – new medicines.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Nature Biotechnology 36, 6 (2018). doi:10.1038/nbt0118-6a
Source: Nature Biotechnology - Category: Biotechnology Tags: News Source Type: research
by Craig Klugman, Ph.D. The FDA has approved the world’s first gene therapy: Luxturna (voretigene neparvovec; AAV2-hRPE65v2) is a one-time intervention that can treat an inherited retinal disease (RPE65-mediated inherited retinal dystrophy). The disease is rare, affecting only 1,000-2,000 people in the U.S. The treatment works by introducing a virus to the eye that contains a healthy copy of the gene that caused the blindness. In a phase 3 trial of 31 subjects (20 intervention; 9 control; 2 withdrew), 65% of patients who received the procedure experienced dramatic improvements in their vision.…
Source: blog.bioethics.net - Category: Medical Ethics Authors: Tags: Clinical Trials & Studies Featured Posts Health Disparities Health Policy & Insurance Health Regulation & Law Pharmaceuticals gene therapy Luxturna Source Type: blogs
Drug-maker Spark Therapeutics lowered the cost of its treatment for a rare form of blindness from $1 million, but at $850,000, the drug is still one of the most expensive gene therapies in the world.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
More News: Biotechnology | Blindness | Gene Therapy | Genetics | Opthalmology