You might remember Gabe Griffin from last summer, when we spotlighted the young advocate and the #HopeForGabe effort, a cross-country bicycle ride raising awareness for Duchenne muscular dystrophy (Learn more about this year’s ride from Maine to Alabama here). At the age of three, Gabe was diagnosed with Duchenne. The disease is the number one genetic killer of boys in the world. Due to the lack of funding and awareness of this deadly disease, boys who are diagnosed are commonly referred to as “The Lost Boys.”

But that’s not stopping Gabe. Gabe and his parents have been encouraged by H.R. 6, the 21st Century Cures Act, and recently made the trip from Birmingham, Alabama, to the nation’s capital to meet with Energy and Commerce Committee Chairman Fred Upton (R-MI). Gabe’s parents spoke about some of the challenges of the disease, and how the 21st Century Cures Act will help Gabe using precision medicine.

Gabe has made quite an impact wherever he travels, whether it’s at the Alabama state capital in Montgomery or the halls of Congress in Washington. As Rep. Martha Roby (R-AL) tweeted after meeting Gabe this month, “Amid all the meetings, hearings & votes, one special visitor put it all in perspective.” Roby also recounts her meeting with Gabe in a column she wrote for the Montgomery Advertiser, “Special visitor makes impact on Capitol Hill.”

Click here to see more photos from Gabe’s meeting with Upton.

Gabe Griffin's family pushes D.C. to pass bill that would help terminally ill Alabama boy

A 10-year-old Shelby County boy who has captured the hearts of Alabamians through his fight with a rare form of muscular dystrophy visited Washington, D.C., with his parents this week to push for a federal bill that may pave the way for stopping the progression of his terminal condition.

Gabe Griffin and his parents, Scott and Traci Griffin, are urging Congress to pass the 21st Century Cures Act, which would allow the Food and Drug Administration to automatically approve so-called "follow-on drugs," or precision medications that use the same technology as previously approved drugs but target a different structure to treat the condition. The bill would not only help Gabe, his father said, but others with rare terminal illnesses with no known cures or treatments.

The 21st Century Cures Act passed the House and is being taken up by the Senate. The Griffins' visit included meetings with House Energy and Commerce Commitee Chairman Fred Upton of Michigan, who is championing the bill, House Majority Leader Kevin McCarthy, R-Calif and Reps. Bradley Byrne and Martha Roby of Alabama.

"[Upton] feels pretty confident that the act is going to pass and I really was shocked how much time he spent with us and how much he knows about Gabe and Duchenne," Griffin said of Upton. He added that Byrne and Roby, aware of Gabe's condition and the boy's difficulty climbing stairs, visited with Gabe and the family instead of making the 10-year-old go to them.

Gabe, a freckled, red-haired boy who enjoys trash talking on the basketball court and playing baseball, suffers from Duchenne, a form of muscular dystrophy caused by a genetic mutation that prevents muscles from using a protein known as dystrophin, resulting in a progressive deterioration of muscle function. His family and advocates have been behind the annual Ride4Gabe, a cross-country bike ride that raises awareness of Duchenne. In the summer, cyclists will embark on an 11-day ride from Maine to Mobile on Gabe's behalf.

Without a cure, Duchenne, which occurs in about one out of every 3,500 live births, will rob Gabe of his ability to walk within the next few years. By the time he graduates high school, it's likely he will need breathing and feeding tubes and have increased difficulty controlling his muscles. Under the most optimistic scenario, Gabe won't reach his 26th birthday.

But there is hope on the horizon, according to Scott Griffin and Michael Staley, a consultant for the family's foundation – Hope For Gabe.

Gabe and his family are hoping that follow-on drugs piggybacking on eteplirsen, a medication in clinical trials that would treat a subset of Duchenne and has shown to be effective, will stop the progression of Gabe's subset. The FDA could rule as early as April 25 to approve eteplirsen.

The clinical trials have shown that the drug takes about six months before it takes effect, meaning Gabe would need the follow-on drug to be developed approved in the next few years for him to maintain his quality of life. His father described the progression of Gabe's Duchenne in the last few years.

"He's starting to get weaker. He struggles with getting off the floor from a sitting position. He gets tired easy. He struggles with stairs. He pretty much can't do stairs. We have to carry him up and down the stairs at our house," Griffin said. "He's starting to ask us for help more and more. The most frightening this is we're seeing it more frequently. When you see it progressing in front of your eyes and there's nothing you can do, that is very, very tough to handle."

The Griffins have shielded Gabe from the reality he faces. But Gabe – and the family – still hold out hope.

"Gabe doesn't know that he's dying, and one day I'm going to have to tell him that and I pray every day that that day doesn't come," Scott Griffin said. "All he knows is that he has weak muscles. He believes that when this drug is approved, hopefully on April 25, he believes he is going to get the drug and it's going to cure him. And it doesn't matter how many times I tell him that is not the case, he believes it. Every one of us [has] hope, and there have been so many godly moments that have happened throughout this journey that have opened doors. And I believe having faith is having courage to step through the door that God has opened."

Click HERE to read the article online.

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