— No price can be suggested as a fair value-based price for eteplirsen or golodirsen because no persuasive evidence yet exists to demonstrate the clinical effectiveness of either drug — — For deflazacort, discounts of at least 73% from its list price would be needed to achieve commonly cited thresholds for cost-effectiveness — — As
A new study, carried out on mice, shows great promise for developing treatments that restore vision in blind people. According to researchers, gene therapy is helping the retina to restructure itself, regain normal light responses and, ultimately regain vision in mice. A paper, published in JNeurosci, outlines the findings and supports the continued development of treatments for
Injured axons instruct Schwann cells to build specialized actin spheres to break down and remove axon fragments, thereby starting the regeneration process Damaged peripheral nerves can regenerate after an injury, for example, following a forearm fracture. Axons, the long projections of neurons that transmit stimuli or signals to other cells, are affected in the case
More than half of new drugs entering the German healthcare system have not been shown to add benefit, argue researchers. More than half of new drugs entering the German healthcare system have not been shown to add benefit, argue researchers in The BMJ today. Beate Wieseler and colleagues at the German health technology assessment agency IQWiG (Institute
Sangamo Therapeutics and Pfizer have reported additional findings from the Phase I/II Alta clinical trial of investigational gene therapy SB-525 to treat patients with severe haemophilia A. SB-525 is made up of a recombinant adeno-associated virus serotype 6 vector (AAV6) that encodes the complementary deoxyribonucleic acid for B domain deleted human Factor VIII (FVIII). During
Resistance to high prices, even with novel payment models Europe’s leading nations should commit to a rapid overhaul of health technology assessment (HTA) and bold initiatives such as new pan-European real world evidence (RWE) frameworks, in order to nurture the nascent cell and gene therapy revolution in the region. That is the conclusion of a
Source STAT If nutritious meals can provide the same kind of benefit as medication, then why don’t health plans cover the cost of medically tailored meals, just as they cover prescription medications? It’s a question my colleagues and I at Community Servings, a nonprofit organization that provides nutritious meals for people with critical illnesses who are
Source Wyss Institute for Biologically Inspired Engineering at Harvard The human microbiome, the huge collection of microbes that live inside and on our body, profoundly affects human health and disease. The human gut flora in particular, which harbor the densest number of microbes, not only break down nutrients and release molecules important for our survival
Source NIH The blood-brain barrier, or BBB, is a dense sheet of cells that surrounds most of the brain’s blood vessels. The BBB’s tiny gaps let vital small molecules, such as oxygen and water, diffuse from the bloodstream into the brain while helping to keep out larger, impermeable foreign substances that don’t belong there. But
Orgenesis, a developer, manufacturer and service provider of advanced cell therapies, announced that the United States Food & Drug Administration (“FDA”) has granted Orphan Drug designation for its Autologous Insulin Producing (“AIP”) cells as a cell replacement therapy for the treatment of severe hypoglycemia-prone diabetes resulting from total pancreatectomy (“TP”) due to chronic pancreatitis (“CP”).
