The Duchenne muscular dystrophy community is in turmoil. The death of a second teenager from liver failure has sparked fear and anger. The teenager died after receiving a gene therapy called Elevidys. This therapy is from a company called Sarepta Therapeutics. The teenager's death has left families in a state of uncertainty and desperation. Kameron Lawson re...

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting condition that strikes young children. It's caused by a problem in a massive gene called DMD. For a long time, doctors have used muscle biopsies to diagnose and track DMD. But these procedures can be risky and uncomfortable. That's where blood tests come in. They're a safer and simpler way to spot signs of ...

A recent event has brought attention to the risks of gene therapy for Duchenne muscular dystrophy. A 16-year-old boy lost his life after receiving Elevidys, a treatment designed to combat this severe condition. This unfortunate incident marks the first known death associated with this particular therapy. The teenager's passing was due to liver failure, a complication...

Inflammation and oxidative stress are big troublemakers when it comes to skeletal muscle diseases. Scientists are now looking at a protein called APE1/Ref-1, which plays a role in DNA repair and controls other proteins, like NF-κB and NRF2. These proteins are often targeted in treatments, but they haven't worked so well. APE1/Ref-1 is like a hidden key that might unl...

Duchenne Muscular Dystrophy (DMD), a severe genetic condition that mainly affects boys, causes muscles to weaken over time. This leads to serious health issues like heart problems or breathing difficulties, and sadly, it can be fatal. Right now, there's no cure, and the usual treatments just help manage symptoms. Enter gene therapy. It targets the real cause of DMD—...