An international workshop of scientists, regulators, and industry experts led by Prof Ivana Barbaric convened to address the risks of genetic mutations in stem cell therapy. Subscribe to our ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
A groundbreaking Cleveland Clinic trial is demonstrating how gene-editing technology could transform the way millions of Americans manage high cholesterol. A Phase 1 clinical trial at Cleveland Clinic ...
Cell and gene therapies are currently niche products, even within specialty pharmacy, drugs that, by most definitions, are ...
A patient in a clinical trial for a Regenxbio gene therapy developed a brain tumor, leading the FDA to place the study under a clinical hold. This pause has implications for another Regenxbio gene ...
Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...
Pediatric-Friendly Formulations: Critical to Maximizing Benefit of Novel Therapeutics Such as Selumetinib for Children With Neurofibromatosis and Other Neoplasms Supported in part by the Clinical and ...
The year 2023 marked a significant milestone for the treatment of sickle cell disease (SCD), with the approval of two gene therapies shown to help reduce the pain experienced by people with this ...
Two papers published in The New England Journal of Medicine highlight the potential of gene editing for treatment of sickle ...
The ethics of using safe gene therapies to improve the health and cognition of Down syndrome children and adults.
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