Setback in gene therapy for Duchenne muscular dystrophy as immune system emerges as key barrier

Setback in gene therapy for Duchenne muscular dystrophy as immune system emerges as key barrier

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a large-scale clinical trial.

Her son’s DMD diagnosis shook her world. As a biotech CEO, it was also an epiphany for better rare disease R&D.

Five years ago, Michelle Werner received her son’s Duchenne muscular dystrophy diagnosis with fear and desperation. Now, as CEO of Alltrna, she’s finding ways to turn those feelings into a better way of treating rare diseases.

Care Gets a Lift: How Kevin Carbone Got Back to the Water with Help from Jett Foundation - Jett Foundation

For the Carbone family, water has always been more than just a place to swim—it’s a source of therapy, joy, and connection. When the COVID-19 pandemic hit in 2020, Kevin, who was just seven years old at the time and beginning to face the physical challenges of Duchenne muscular dystrophy, could no longer access his usual therapeutic pool. The local town pool was too cold, and closures due to COVID-19 made warm water therapy inaccessible. So, the…

Setback in Gene Therapy for Duchenne Muscular Dystrophy as Immune System Emerges as Key Barrier

A new paper, published in Gene Therapy, raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a large-scale clinical trial.