Spark reveals the price tag for its groundbreaking gene therapy treatment

The Philadelphia company will offer outcomes-based rebates and the possibility for installment payments to help ensure access to the one-time therapy that treats a condition that otherwise leads to blindness.
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news

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Matthew Bishop was told there was no treatment that could save his vision. But now scientific breakthroughs in gene therapy have given him, and others, hopeIn his office in Oxford ’s John Radcliffe hospital, Prof Robert MacLaren sits upright, his back as straight as a soldier’s, and tells me about the lowest point in his 20-yearcareer. It was the rejection, many years ago, of his grant application for a project investigating how gene therapy might treat conditions causing blindness. “It was completely panned by the reviewers,” he says. “We were told ‘There’s no way it’s ever ...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Genetics Parents and parenting Biology Science Family Life and style Source Type: news
CONCLUSIONS: In children complaining of night blindness, a PDE6Β-associated RP needs to be taken into consideration. Apart from helping patients with optical aids, such as polarizing filters or magnification, a specific diagnosis is especially important in view of emerging genetic treatment options. In particular, in RP patients with a PDE6Β mutation, a phase I/II study is currently ongoing (https://clinicaltrials.gov/ct2/show/NCT03328130). PMID: 30646425 [PubMed - as supplied by publisher]
Source: Klinische Monatsblatter fur Augenheilkunde - Category: Opthalmology Tags: Klin Monbl Augenheilkd Source Type: research
AbstractInherited retinal diseases (IRDs) are a group of rare, heterogenous eye disorders caused by gene mutations that result in degeneration of the retina. There are currently limited treatment options for IRDs; however, retinal gene therapy holds great promise for the treatment of different forms of inherited blindness. One such IRD for which gene therapy has shown positive initial results is choroideremia, a rare, X-linked degenerative disorder of the retina and choroid. Mutation of theCHM gene leads to an absence of functional Rab escort protein 1 (REP1), which causes retinal pigment epithelium cell death and photorec...
Source: Pharmaceutical Research - Category: Drugs & Pharmacology Source Type: research
This study suggests that advantages and disadvantages vary by environment and diet, however, which might explain why evolution has selected for multiple haplogroups rather than one dominant haplogroup. This is all interesting, but none of it stops the research community from engineering a globally better-than-natural human mitochondrial genome, and then copying it into the cell nucleus as a backup to prevent the well-known contribution of mitochondrial DNA damage to aging. Further, nothing stops us from keeping the haplogroups we have and rendering the effects of variants small and irrelevant through the development...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Discussion of Mitochondrial Hormesis as an Approach to Slow Aging Cornelis (Cees) Wortel, Ichor Therapeutics Chief Medical Officer, on Rejuvenation Research and Its Engagement with the Established Regulatory System An Interview with a Programmed Aging Theorist An Interview with Reason at the Life Extension Advocacy Foundation An Interview on Mitochondrial Damage and Dysfunction in Aging An Interview with Vadim Gladyshev on Research into the Causes of Aging An Interview with Jim Mellon, and Update on Juvenescence A Lengthy Interview with Aubrey de Grey of the SENS Research Foundation An Interview with Peter de Keize...
Source: Fight Aging! - Category: Research Authors: Tags: Healthy Life Extension Community Source Type: blogs
This article reviews gene therapy as it can be applied to inherited retinal dystrophies, provides an update of results from recent clinical trials, and discusses the future prospects of gene therapy and genome surgery.
Source: Molecular Diagnosis and Therapy - Category: Molecular Biology Source Type: research
MacLaren Inherited retinal degenerations are the leading cause of blindness in the working population. X-linked retinitis pigmentosa (XLRP), caused by mutations in the Retinitis pigmentosa GTPase regulator (RPGR) gene is one of the more severe forms, and female carriers of RPGR mutations have a variable presentation. A retrospective review of twenty-three female RPGR carriers aged between 8 and 76 years old was carried out using fundoscopy, autofluorescence imaging (AF), blue reflectance (BR) imaging and optical coherence tomography (OCT). Confirmation of the genetic mutation was obtained from male relatives or Sanger...
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Article Source Type: research
DISCUSSION: In the text, we first introduce the pathological development of corneal disease in the aspects of neovascularization and inflammation. We summarize how MSCs become an ideal candidate in cell therapy for treating injured corneal, focusing on cell biology, property and features. We provide an updated review of gene-based therapies in animals and preclinical studies in the aspects of controlling target gene expression, safety and efficacy. Gene transfer vectors are potent to induce candidate protein expression. Delivered by vectors, MSCs are equipped with certain characters by expressing protein of interest, which...
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Tags: Curr Gene Ther Source Type: research
(Children's Hospital of Philadelphia) Children's Hospital of Philadelphia celebrates a pivotal moment in medicine: approval by the European Commission of LUXTURNA ® (voretigene neparvovec), the first and only gene therapy for patients with an inherited retinal disease, last month. This also makes LUXTURNA the first gene therapy for a genetic disease that has received regulatory approval in both the US and European Union.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
Novartis's price-setting muscle is about to be tested after it won European Union approval for a blindness-fighting gene therapy whose $850,000 list cost in the United States has been labeled too expensive by some groups.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
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