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Genetic Linkage

FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

Dystrophin protein (green) is deficient in DMD, and muscles fall apart.

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. Boys 6 years and older can take Duvystat, to slow the course of the illness. FDA classifies it as a "nonsteroidal treatment" – not a gene therapy, but it affects gene expression.

 

To continue reading, go to DNA Science, where this post first appeared.

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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It, I predicted that the technology would soon expand well beyond the rare disease world.

 

I was overoptimistic. Gene therapy clearly hasn't had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases. We're still learning possible outcomes of sending millions of altered viruses into a human body. Can they deliver healing genes without triggering an overactive immune response?

 

A report in the September 28, 2023 The New England Journal of Medicine describes a young man with Duchenne Muscular Dystrophy (DMD) who died just days after receiving gene therapy. The details are disturbingly reminiscent of the famous case of Jesse Gelsinger, who died from a ferocious immune response to experimental gene therapy in September 1999.

 

To continue reading, go to DNA Science, where this post first appeared.

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Genetics in 2016: Breakthrough, Breakdown, and Bridge

I dislike end-of-year "best of" lists, especially the "breakthroughs" that imply scientific discoveries and medical advances emerge from out of nowhere. Often they're the product of PR machines that select and then catapult certain research findings into the news releases that dictate the headlines.

WHAT MAKES SCIENCE NEWS?
Much of science news is released to journalists ahead of time (embargoed) so that we can investigate background and conduct interviews. This year, the Food and Drug Administration began offering news even earlier to select media outlets.  Read More 
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