Progress is Being Made in Duchenne Muscular Dystrophy Gene Therapy

Researchers at the University of Pennsylvania are working on a new potential gene therapy for Duchenne muscular dystrophy (DMD). DMD is a rare form of muscular dystrophy that primarily affects males.

The condition is caused by a gene mutation which stops the body from producing a critical muscle-building protein. By correcting this mutation, symptoms of the disease may be diminished.

Yuva Gambhir

Yuva Gambhir is an 18-year-old DMD patient who lives in Philadelphia. He is an unofficial patient ambassador, and a strong advocate for the advancement of this treatment.   

He candidly speaks to life before illness, and life after his symptoms developed. Although he’s adapted well, he explains how it was hard growing up not being able to play with his friends in the same way other kids could. He couldn’t keep up physically, which affected how he was able to participate.

Gambhir is excited about the potential of gene therapy for DMD and is anxious to see where this research goes. He hopes it will help other patients experience youth differently than he did.

About the Research

The research is personal for the team at UPenn. The lead researcher on the project is named Stedman and two of his brothers passed away from DMD. This personal connection has made him even more determined to find a cure for this rare disease. Co-author Leon Morales shares a similar sentiment. He himself has a more mild form of muscular dystrophy and he’s excited to see how this research could affect his own community.

Currently the gene therapy has been tested in animals. With FDA approval, they’ll be able to move on to human trials.

The Ultimate Dream

The ultimate dream with this therapy is that as soon as a child is diagnosed with the condition, they can be administered the treatment and never experience a single symptom.

This would change the lives of not only patients but their families, allowing them to live a long and normal life.

You can read more about this potential gene therapy here.


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