Yale School of Medicine developing first-ever treatment for Duchenne Muscular Dystrophy

Health News

NEW HAVEN, Conn. (WTNH) — Terry Horgan was born with Duchenne Muscular Dystrophy 24 years ago. He is among 15,000 people living with Duchenne.

Rich Horgan, Terry’s brother, told News 8, “Duchenne Muscular Dystrophy is a fatal progressive neuromuscular condition that impacts boys and oftentimes is 100% fatal in boys today.”

Horgan says the disease comes in many different forms and treating the disease with a one size fits all approach would be very difficult.

To save Terry’s life and thousands of others, Horgan says they are, “trying to develop an approach that has never been tried before.”

Terry’s older brother Rich Horgan founded the non-profit Cure Rare Disease.

He says, “Cure Rare Disease is the only one funding this and really it’s because we wanted to take a radically different approach to try and treat disease.”

Related: Duchenne muscular dystrophy: Developing therapy gives Branford family hope

Yale researchers led by Monkol Lek are among those at six institutions pioneering what could be a life-saving treatment.

Lek said, “What led me to start working on this, knowing your genetic disease is a great thing but knowing how to fix it is even better.”

They want to fix it by essentially correcting the patient’s mutated DNA in the lab.

“The technology is CRISPR; it allows us to very precisely edit a place in the human genome,” Lek said. “We don’t know if it’s going to reverse it but what we do know is by putting something back that wasn’t actually being produced and what’s causing the disease, that it would at least halt the disease.”

Lek added, “I am very optimistic that it will work, mainly because when you show something happening on human cells, you can be optimistic that its targeted and it will work on a personal level.”

The results are so promising that animal testing to determine how safe and effective it is starts in January. If all goes well, Terry will be the first patient to undergo treatment.

Horgan said, “This approach is especially interesting because it will be the first in-man application of a CRISPR activation technology to our knowledge ever.”

Web Extra: Research continues to find treatment for Duchenne muscular dystrophy

“[Terry] like many of the boys and young men impacted by this disease are desperate for hope,” Horgan added.

A radical approach needed — Rich Horgan says so that “today’s patients can see tomorrow’s cures.”

They are working closely with the FDA on this study.

For more information on Cure Rare Disease, go to cureraredisease.org.

Want to support its mission? You can donate on the website or text CureOneCureAll to 44-321.

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