TAKING NOTES:

Just a couple weeks ago, right about the end of 2007, a small bio-tech company in the Netherlands known as Prosensa made a startling announcement.

After conducting trials, the drug maker believes it has discovered a therapy that could provide those who suffer from Duchenne muscular dystrophy a chance at living a full and complete life.

The announcement didn’t get a lot of ink in these parts. There was small writeups in the New England Journal of Medicine, the Los Angeles Times, the New York Times and blurbs in some medical publications.

But right here in Newport Beach, Debra Miller was most likely beaming with pride and teaming with hope.

You see it was last May in this space, that I told you about Miller and the organization she founded, CureDuchenne — an organization born, quite literally, to save the life of her son, who has the disease.

She told me then about some of the researchers she is funding and how she didn’t believe it was going to take that many millions to put a serious dent in this disease.

And one of those researchers she funded, with a grant to the tune of $1.3 million five years ago, was Prosensa.

While $1.3 million may not seem like enough to cure a disease, the money came when Prosensa needed it most, filling a gap for the company at a crucial time and when it was in need of a human trial, Miller said.

“The most important thing about this is it is a whole new technology,” Miller told me. “By this being successful it really opens the door. It’s a huge breakthrough and we are really proud that we had the guts to go forward with it.”

I called Gerard Platenburg, the CEO of Prosensa, and asked him just how big a role this little Newport Beach-based non profit played in his company’s amazing breakthrough.

“They were kind of catalyst so to speak,” he said. “Obviously we have been working with several groups. But they have been instrumental for us to continue to grow. They were very motivating and very helpful in raising money. Most importantly we are now at the stage where we can test this therapy in trials.”

Miller said she is just happy she took the chance on Prosensa, which now has some $18 million worth of financing at its fingertips.

“So many of the academic researchers are just so focused on tinkering and perfecting things,” she said. “Prosensa kind of shook things up because someone went to trial with this drug.”

How this drug works is way too complicated for me to understand, much less explain to you the readers. It has something to do with replacing missing proteins and genes and things like that.

And it should be noted that right now the treatment has a price tag of $250,000 for a year’s worth of therapy. But Miller believes in time the cost will be substantially less, making access to the therapy more widely available to boys who otherwise would be in wheelchairs by their teens and unlikely to live beyond their 20s.

It’s Miller’s hope that Duchenne muscular dystrophy becomes much like Type 1 diabetes where “yeah you have it but you are living with it.”

Prosensa’s CEO seems optimistic that day is not far off.

“I feel that with the approach we have taken that we are really moving to that point,” Platenburg said. “It will be a lifelong treatment. But a lifelong treatment, if we are successful, will allow a patient to live a life that is near normal.”

Miller, who founded CureDuchenne because she didn’t like the approach other muscular dystrophy organizations took toward research and cures, said just the Prosensa breakthrough alone makes her efforts successful.

But she’s not sitting on her heels any time soon.

“We need to keep the pressure on and we need to keep funding,” she said. “Our goal is to get people to realize that small can be mighty and to keep the pressure on. We want to save our kids.”

To learn more about this disease or how to get involved in CureDuchenne, go to their website at www.cureduchenne.org.

TONY DODERO is the director of news and online. He can be reached at (714) 966-4608 or at tony.dodero@latimes.com.