Developing Treatments for Blindness: Gene Therapy Shown to Restructure Retinas

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

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First step to induce self-repair in the central nervous system

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

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No evidence of added benefit for most new drugs entering German healthcare system

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

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Sangamo and Pfizer report data for haemophilia A gene therapy

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

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Europe urged to adapt faster to gene therapy challenges

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

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Medically tailored meals save lives. Health plans should cover their cost

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

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Microbiome Physiology Can Now Be Studied In Vitro Using Organ Chip Technology

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

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Orgenesis Granted FDA Orphan Drug Designation for Autologous Insulin Producing Cells in the Treatment of Severe Hypoglycemia-Prone Diabetes Resulting from Total Pancreatectomy

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”).

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