Medical industry unites for disease cure

Some of the country’s top medical researchers arrived in Newport Beach on Thursday to help find a cure for a fatal genetic disease that strikes young boys.

The Newport Beach-based nonprofit CureDuchenne is hosting its first Duchenne Muscular Dystrophy Summit at the Newport Beach Marriott this week to discuss the latest developments in medical research on the disease.

“I’m very humbled, first of all, to have this level of top researchers in the world giving up their weekend to come out here,” said mom and CureDuchenne co-founder Debra Miller.

“I’m very grateful to them — my fantasy at the end of the meeting is to be able to say ‘cool, we know exactly what the first projects are that need funding.’ ”

Debra and husband Paul Miller founded CureDuchenne in 2003 after their now junior-high-school-aged son was diagnosed with the disease.

Duchenne muscular dystrophy attacks and weakens the muscles. Historically, most boys diagnosed with the disease do not survive into adulthood. The illness affects more than 1 in 3,500 boys worldwide and is the most common and deadly form of muscular dystrophy.

Muscle disease and drug development experts from all over the country will give the Millers suggestions on what medical research to help fund.

Over the past seven years, CureDuchenne has provided millions of dollars in funding for Duchenne muscular dystrophy research and has helped bring three treatments to the human clinical trial phase.

The Newport Beach summit is being sponsored by the Genzyme Corporation.

“Debra really assembled some of the top people in the world who are working on this disease,” said Dr. Eric Hoffman, a medical researcher from Children’s National Medical Center in Washington, D.C. “More and more things are entering clinical trials for the first time.”

Five years ago, there were no treatments for Duchenne muscular dystrophy in clinical trials, Hoffman said, a period of time that he equates to the “dark ages.”

CureDuchenne has been instrumental in providing money and support for moving some experimental treatments along, he said.

The summit will culminate with a breakfast at 8 a.m. Saturday at the Marriott, 900 Newport Center Drive.

Some of the top medical experts in the country will be at the breakfast to talk about and answer questions about the latest Duchenne muscular dystrophy research.

Tickets cost $25 and may be bought at breakfastwithdmdexperts.eventbrite.com/.