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Here Come More Gene Therapies -- And More Pricing Debates

Gene therapies like Spark Therapeutics'upcoming Luxturna and Novartis'FDA-approved Kymriah are raising concerns about the rising cost of health care, leading some experts to suggest new ways to price treatments based on the benefits they offer.
Source: Forbes.com Healthcare News - Category: Pharmaceuticals Authors: Tags: NASDAQ:ONCE Source Type: news

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CONCLUSION: LPC conditioning prior to LV vector delivery, substantially enhanced the level of conducting airway gene expression after a single gene vector delivery. These results extend the previously established effectiveness of this protocol for producing gene expression in the nasal airways to the lung airways, the primary site of deleterious pathophysiology in CF individuals. PMID: 29236544 [PubMed - as supplied by publisher]
Source: Experimental Lung Research - Category: Respiratory Medicine Tags: Exp Lung Res Source Type: research
22 –25 January 2018, Miami, United States
Source: Scientific and Medical Events on Global Events List - Category: Science Source Type: events
Development of an adenovirus vector vaccine platform for targeting dendritic cells, Published online: 15 December 2017; doi:10.1038/s41417-017-0002-1Development of an adenovirus vector vaccine platform for targeting dendritic cells
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
Alzheimer's disease (AD) is a progressive neurodegenerative disease characterized by synaptic dysfunction and accumulation of abnormal aggregates formed by amyloid‐β peptides or phosphorylated tau proteins. Accumulating evidence suggests that alterations in the buffering capacity of the proteostasis network are a salient feature of AD. The endoplasmic reticulum (ER) is the main compartment involved in protein folding and secretion and is drastically affected in AD neurons. ER stress triggers the activation of the unfolded protein response (UPR), a signal transduction pathway that enforces adaptive programs to recove...
Source: FEBS Journal - Category: Research Authors: Tags: State ‐of‐the‐Art Review Source Type: research
A gene-therapy trial by London researchers has seen remarkable success in finding a long-term cure for hemophilia A.
Source: CNN.com - Health - Category: Consumer Health News Source Type: news
Jake Omer, 29 from Billericay, Essex, who took part in the Bars Health NHS Trust trial, revealed the gene therapy changed his life and that he now has 'hope for the future'.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
A CURE for haemophilia is one step closer after “mind-blowing results” from a gene therapy trial.
Source: Daily Express - Health - Category: Consumer Health News Source Type: news
A 'cure' for haemophilia is one step closer, following results published in the New England Journal of Medcine of a groundbreaking gene therapy trial led by the NHS in London. Clinical researchers at Barts Health NHS Trust and Queen Mary University of London have found that over one year on from a single treatment with a gene therapy drug, participants with haemophilia A (the most common type) are showing normal levels of the previously missing protein, and effectively curing them.
Source: World Pharma News - Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
How pioneering gene therapy has given a haemophilia patient a new lease of life.
Source: BBC News | Health | UK Edition - Category: Consumer Health News Source Type: news
Doctors use gene therapy to correct the defect that causes haemophilia A.
Source: BBC News | Health | UK Edition - Category: Consumer Health News Source Type: news
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