The results of a first-in-human clinical trial of gene therapy to treat a common cause of genetic blindness have shown partial reversal of sight loss in some patients. X-linked retinitis pigmentosa, caused by mutations in RPGR gene, is the most common cause of blindness in young people. The inherited mutations lead to degeneration of light… Read More »
A new study provides insights on why some people may be more resistant to Alzheimer’s disease than others. The findings may lead to strategies to delay or prevent the condition. The study was led by investigators at Harvard-affiliated Massachusetts General Hospital (MGH), in collaboration with the University of Antioquia, Schepens Eye Research Institute of Massachusetts Eye… Read More »
We live in an incredible era of progress in human health. Advances in cell and gene therapies are beginning to yield powerful new treatments for some of the most devastating illnesses. With many of these new therapies we are no longer combating diseases over time, but potentially curing them in a single treatment. This is… Read More »
(Reuters) – Novartis AG, which this week announced positive interim trial results for its experimental gene therapy for spinal muscular atrophy, on Friday said investigation is underway into whether a second trial death could be related to the treatment. The logo of Swiss drugmaker Novartis AG is seen at its headquarters in Basel, Switzerland January… Read More »