LND topic: muscular-dystrophy page 1

muscular dystrophy

When Radiation Hits Weak Muscles: New Findings in a Rare Childhood Disorder

April 22, 2026

Researchers have uncovered fresh clues about how muscle cells react when exposed to radiation in Duchenne muscular dystrophy (DMD). Unlike typical muscle loss seen in the disorder, these cells show an unusual sensitivity to radiation that may speed up damage. Boys with DMD, who make up almost all ca

health

A New Hope for Duchenne Patients: ELEVIDYS Shows Promise

January 26, 2026

Duchenne muscular dystrophy is a tough disease that makes muscles weak over time. Kids with this condition often struggle with simple movements like standing up or walking. But there's some good news! A treatment called ELEVIDYS is showing positive results in a big study called EMBARK. The study lo

health

A Drug Company and the FDA in a Heated Dispute Over Patient Safety

July 20, 2025

In a surprising turn of events, Sarepta Therapeutics has chosen to defy the FDA's request to stop distributing its gene therapy drug, Elevidys. This decision comes after three patient deaths linked to the treatment. The FDA has expressed serious concerns about the safety of the drug and has asked Sa

health

A Second Death Shakes Duchenne Community

June 17, 2025

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 famil

health

Blood Tests for Muscle Disease: The Future of Duchenne Diagnosis

May 29, 2025

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 b

health

Teen's Death Raises Questions About Duchenne Gene Therapy

March 18, 2025

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 the

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Exploring APE1/Ref-1: A New Approach to Fight Duchenne

December 27, 2024

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 h

health

Gene Therapy: A New Hope for Duchenne Muscular Dystrophy?

December 25, 2024

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 ma