Dear Duchenne Community, We want to provide you with an update on the progress of SolidBiosciences IGNITE DMD Phase I/II clinical trial for our investigational microdystrophin gene transfer, SGT-001. Since we communicated preliminary clinical data in February, we advanced the study to evaluate SGT-001 at a higher dose in the second cohort of patients. As
GRAIL, Inc., a healthcare company whose mission is to detect cancer early, when it can be cured, today announced that its multi-cancer test has been granted Breakthrough Device designation from the U.S. Food and Drug Administration (FDA). The investigational blood test is in development for the early detection of multiple cancer types in individuals aged
Machine learning algorithm predicts sudden death in heart failure patients for the first time Artificial intelligence (AI) has shown promise to select heart failure patients for expensive treatments to prevent lethal arrhythmias, reports a study presented today at ICNC 2019. (1) The study is the first to use a machine learning algorithm to predict sudden
Last year, the birth of the first genetically modified babies shook the world. The German Ethics Council now presents a comprehensive ethical investigation into possible interventions in the genome of human embryos or germ cells. The Council does not deem the human germline to be inviolable. It does, however, consider germline interventions to be ethically
Despite the binary thinking of progressive scientists on the one hand and prophets of doom on the other, science doesn’t tend towards good or evil. Any experiments scientists can do, they will. This is a positive thing, because who is a position to claim omniscience and to forbid it? In fact, it’s human beings who
**For the first time, researchers have developed a non-intrusive way to generate large quantities of stem cells using only a small amount of blood** **The stem cells can repair cells damaged as a result of vascular diseases, which has the potential to prevent blindness and reverse the need for amputations** A discovery by researchers at
The Alliance for Regenerative Medicine (ARM) today released its most recent quarterly data report, offering an in-depth look at cell therapy, gene therapy, tissue engineering, and broader global regenerative medicine sector trends and metrics in the first quarter of 2019. By further curating information provided by ARM’s data partner Informa, the quarterly data report details
A discovery opens the possibility of one day restoring loss of vision by activating the retina’s ability to regenerate. Researchers at Baylor College of Medicine, the Cardiovascular Research Institute and the Texas Heart Institutereveal in the journal Cell Reports that although the mammalian retina – a layer of specialized nerve cells that mediates vision and is located on the back
As a lung transplant surgeon, Matt Bacchetta has watched countless patients wait for an organ, only to not get one. About 80 percent of donor lungs are not in good enough to shape to use. Now Bacchetta and a team of researchers from Columbia University in New York City have shown that they can repair
For the first time, scientists have used genetically modified viruses to treat a patient fighting an antibiotic-resistant infection. Isabelle Carnell-Holdaway, 17, began the experimental treatment after doctors lost all hope. She was struggling with a life-threateninginfection after a lung transplant. With the new treatment, she has not been completely cured. But the Faversham, England, teenager has
