Gene therapy shows promise in initial trial for patients with childhood blindness
Research News Release
EurekAlert! provides eligible reporters with free access to embargoed and breaking news releases.
Eligibility GuidelinesEurekAlert! offers eligible public information officers paid access to a reliable news release distribution service.
Eligibility GuidelinesEurekAlert! is a service of the American Association for the Advancement of Science.
A new gene therapy for one of the most common forms of congenital blindness was safe and improved patients' vision, according to initial data from a clinical trial
Researchers at the Medical University of South Carolina have developed a new digital platform called Research Integrated Network of Systems (RINS). This platform enables information about clinical studies to be more easily shared across disparate systems. RINS provides a much-needed means to track clinical studies and measure their success. The decentralized program allows users from different disciplines or administrative offices to utilize the system that works best for them while maintaining a comprehensive reporting mechanism.
The preliminary trial results of a novel radiopharmaceutical for PET imaging of inflammation developed at the University of Turku, Finland, have been published. The compound, which targets the vascular adhesion protein 1 (VAP-1) that regulates inflammatory cell traffic, is the first radiopharmaceutical that has been developed completely in Finland and has advanced to clinical trials. In the study that started with healthy volunteers, the radiopharmaceutical was found to be well tolerated and safe.
Despite guidelines recommending de-escalation of treatment among older women with early breast cancer, many continue to be over treated.
The results of the first clinical trial of a new vaccine for a neglected tropical disease have demonstrated that it is safe and induces immune responses in patients with the infection.
Real-word evidence is suggesting, for the first time, the most beneficial treatment courses that could help extend the lives of patients with metastatic non-small cell lung cancer.
Before humans can benefit from new drug therapies and nutritional additives, scientists test their safety and efficacy in animals, typically mice and rats. But, as much as they've done for biomedical research, rodents aren't always the best research model for studies on neonatal brain development and nutrition. That's where pigs can play an important role.
Scientists at Sanford Burnham Prebys Medical Discovery Institute have shown that two existing drug candidates--JAK inhibitors and Mepron--hold potential as treatments for a deadly acute myeloid leukemia (AML) subtype that is more common in children. The foundational study, published in the journal Blood, is a first step toward finding effective treatments for the hard-to-treat blood cancer.
Psilocybin, the active compound in magic mushrooms, may be at least as effective as a leading antidepressant medication in a therapeutic setting.
The April 2021 issue of JNCCN--Journal of the National Comprehensive Cancer Network publishes new research from Memorial Sloan Kettering Cancer Center (MSK) and Gustave Roussy Institute, which suggests that baseline brain imaging should be considered in most patients with metastatic kidney cancer.