Gene Therapy
Sickle cell gene therapy ‘benefits far outweigh the risks,’ FDA adviser says
An FDA panel appeared comfortable with theoretical safety concerns during an advisory committee meeting considering the risks vs. benefits of what would be the first gene therapy for the treatment of sickle cell disease.
Novel gene therapy shows preliminary efficacy for glioblastoma
A novel oncolytic virus gene therapy extended survival among a small cohort of patients with high-grade glioblastoma, according to study results published in Nature.
‘One giant leap for HCM’: Gene therapy may reverse hypertrophic cardiomyopathy
Despite advances made for the treatment and management of hypertrophic cardiomyopathy, the condition remains a debilitating one for many patients, who are often undiagnosed until the disease has progressed.
VIDEO: Highlights in neovascular AMD from ASRS
SEATTLE — In this video, Aleksandra V. Rachitskaya, MD, FASRS, discusses some of the key highlights in neovascular age-related macular degeneration from the ASRS annual meeting.
National Sickle Cell Awareness Month: New insights into gene therapy, pain management
September is National Sickle Cell Awareness Month.
Novel gene therapy offers ‘clinically meaningful improvement’ in sickle cell disease
The first three patients who received an investigational gene therapy for sickle cell disease experienced dramatic reductions in complications related to sickle cell disease, according to results of a first-in-human study.
AMD highlights from ASRS: Positive real-world faricimab data, gene therapy and more
The American Society of Retina Specialists annual meeting featured study results and research in age-related macular degeneration.
Despite safety concerns, gene therapies clinically beneficial for sickle cell disease
Evidence suggests two investigational gene therapies provide net health benefits compared with standard approaches for sickle cell disease, according to a revised evidence report from Institute for Clinical and Economic Review.
Neuro-ophthalmology enters new era of treatment
Neuro-ophthalmologists have a lighthearted attitude about the perception of their specialty because, for many years, neuro-ophthalmology was strictly a diagnosing specialty.
Base-edited gene therapies ‘may be superior’ for sickle cell disease
Genomic alteration using adenine base editors demonstrated the most potency in restarting fetal hemoglobin expression in cells of patients with sickle cell disease, study results showed.