The trial, which is ongoing in the U.K. and New Zealand, has been on hold in the U.S. since late last year as the FDA sought more details from Verve. Verve Therapeutics plans to soon start testing a gene editing therapy for heart disease in the U.S. after federal regulators lifted an order that had
Researchers are testing an approach that involves replacing a mutated gene in the inner ears of children with severe hearing loss. Two companies have launched clinical trials to see if they can restore hearing to children with a rare type of genetic deafness. Akouos and Decibel Therapeutics, both based in Boston, are testing experimental therapies in
Glioblastoma (GBM), an aggressive brain cancer, is notoriously resistant to treatment, with recurrent GBM associated with survival of less than 10 months. Immunotherapies, which mobilize the body’s immune defenses against a cancer, have not been effective for GBM, in part because the tumor’s surrounding environment is largely impenetrable to assaults from the body’s immune system.
Incredibly detailed cell maps help pave the way for new generation of treatments A group of international scientists have mapped the genetic, cellular, and structural makeup of the human brain and the nonhuman primate brain. This understanding of brain structure, achieved by funding through the National Institutes of Health’s Brain Research Through Advancing Innovative Neurotechnologies® Initiative, or
Brazilian researchers and collaborators in Germany and the US compiled data from 195 clinical trials conducted in 30 countries between January 2020 and December 2021. The findings are promising, although the authors stress the need for enhanced controls in the making of the products used in cell therapy. Their findings are reported in an article
Action FDA approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Qalsody is an antisense oligonucleotide that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. The approval was based on a reduction in plasma neurofilament light (NfL), a blood-based
Today, the U.S. Food and Drug Administration approved Omisirge (omidubicel-onlv), a substantially modified allogeneic (donor) cord blood-based cell therapy to quicken the recovery of neutrophils (a subset of white blood cells) in the body and reduce the risk of infection. The product is intended for use in adults and pediatric patients 12 years and older
A joint research team of POSTECH, Kyungpook National University, and Korea University Anam Hospital developed an osteogenic barrier coating material that maximizes the effect of guided bone regeneration (GBR) for implant placements. One of the key factors of success in a dental implant is the condition of the periodontium around the implant. A higher long-term
Key Takeaways In people with type 1 diabetes, the body’s immune system attacks and destroys insulin-producing β cells that control blood glucose levels and are part of a group of cells in the pancreas called pancreatic islets. In research published in Cell Reports Medicine, a team led by investigators at Massachusetts General Hospital (MGH), a founding member
A minimally invasive treatment that injects allograft disk tissue into the spine to relieve pain associated with degenerative disk disease provides significant improvement in pain and function over a sustained period, according to new research to be presented at the Society of Interventional Radiology Annual Scientific Meeting in Phoenix, March 4–9. The treatment, known as viable disk
