Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...
Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...
A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the ...
Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways ...
Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...
This sponsored article was provided by a partner and is not editorial content from Los Angeles Magazine or the Engine Vision Media Network. Every cell in your body holds a unique genetic code within ...
The agency will consider a submission that includes pooled data from ongoing studies, a decision analysts viewed as a notable, additional sign of regulatory flexibility for gene therapies.
Treatment introduces an expansion of the scope of gene therapies that might lead to similar treatments for neurodegenerative ...
The AMT-130 therapy is delivered into the body inside a harmless virus, which serves as a delivery truck for the microRNA. (These types of viruses are commonly used in gene therapy.) Getting the ...
JCR presented non-clinical data that demonstrate the ability of its proprietary JUST-AAV platform to achieve efficient ...
Within minutes of KJ Muldoon’s birth, doctors knew there was something very wrong. Five weeks premature, his little arms went rigid when lifted and shook oddly on the way back down. An attentive ...
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