"If I do the countdown, my son has maybe 5 to 7 years to live," Charaine Woods tells PEOPLE
Credit: Courtesy Charaine Woods

When Charaine Woods’s son was diagnosed with Duchenne muscular dystrophy, a rare and fatal muscle disorder, it was the first of many heartbreaks.

At each appointment with an occupational therapist to help her 9-year-old son Damon cope with the disease that weakens muscles until sufferers lose the ability to walk and breathe, ultimately proving fatal in the early 20s, Woods was faced with concrete evidence of his rapid decline.

“Each time the doctor would say how much weaker he was getting. It was just like getting the diagnosis over and over again,” the San Diego Heights, California, mom tells PEOPLE of her son’s appointments. “Just going in the room was heartbreaking.”

Then, when Damon was 11, he enrolled in a clinical trial for the first drug to treat the progressive and fatal disease. Within one month, Woods says, her son had stopped falling – an instance that had occurred so frequently that anytime anything dropped in the family’s home Damon would shout “I’m okay!” to reassure his mother.

“Then we went to see the occupational therapist and she said, ‘I can’t believe this, he’s getting stronger,’ ” Woods, 35, recalls.

Now, the family’s fate hangs in the balance of a Food and Drug Administration (FDA) decision, expected in early January, about whether or not to approve the drug called drisapersen that Woods and other moms say has dramatically slowed the progress of their sons’ devastating disease.

A Single Hope

Drisapersen would treat approximately 13 percent of Duchenne patients whose disease stems from a flaw in the gene that produces dystrophin – a crucial protein that protects muscle fibers. The drug works through a process called “exon skipping,” which enables the body to “skip” over that genetic defect, explains Dr. Craig McDonald, who leads a clinical trial of the drug in California.

While the moms call the drug a “miracle,” the FDA voiced concerns in a November briefing that cited “life threatening” safety risks and “contradictory” efficacy data.

Moms like Woods argue that these “safety risks” noted by the FDA pale in comparison to the inevitable outcome their sons face with the disease.

“If I do the countdown, my son has maybe 5 to 7 years to live,” Woods says. “This is his only lifeline. There are no other treatments out there. This is it. I don’t have time on my side to continue to wait for [the FDA] to continue debating about this.”

Woods learned how little time she and her son had when an interruption in Damon’s clinical trial caused an abrupt halt in his treatment.

After making significant improvements, Damon sprained his toe while he was off the drug – he’s been wheelchair bound ever since.

“My son lost a part of his life when that treatment stopped,” Woods says through tears. Now that treatment has resumed, she worries about what else Damon stands to lose if the FDA rejects the drug.

“I know other little boys who have this disease and are not on the drug and they cant even wash their own hair, they cant hug their parents, this disease is very progressive and rapid and it gets ugly very fast,” she says. “If treatment stops Damon will lose the only independence he has.”

An Early Start

Tonya Carlone of Maple Valley, Wash. watched her cousin slowly die of Duchenne in the 1980s, so she knew all too well what to expect when her son Gavin was diagnosed at 18 months.

“After seeing what my cousin went through, I was devastated beyond belief to get this diagnosis,” Carlone, 44, says. “I was told by Gavin’s doctor not to count on a cure in his lifetime.”

Still, the mom of three kept looking for options, ultimately getting Gavin into a clinical trial for drisapersen in British Columbia. One year after beginning treatment at age 5, she says, Gavin was “doing things I never thought would be possible.”

Now, at 9, Gavin is able to ride a bike, play on a soccer team and keep up with his healthy peers. Dr. McDonald says he’s never seen anything like the young boy’s progress.

“I’ve been treating Duchenne patients for 25 years and Gavin looks completely different from anyone I’ve ever treated,” he tells PEOPLE.

Carlone believes drisapersen is the only thing that will save her son from the rapid decline that took her cousin’s life. So she’s doing everything she can to sway the FDA’s decision. She has joined Woods in testifying before FDA advisory panels, reached out to media and even started an online petition that has been signed by more than 165,000 people.

‘It’s devastating waiting for the FDA to make a decision,” Carlone says. “It’s very scary to think they might not approve this medication that is dramatically altering my son’s life.”

A Big Decision

Carlone and others in the Duchenne community argue that the FDA’s review is limited and fails to note the drug’s long-term effects. In November, the FDA briefing noted “while there may be some evidence suggestive of efficacy of drisapersen, the evidence is inconsistent and in some cases contradictory, and does not reach the level of substantial evidence.”

McDonald says the FDA review fails to take into account the long-term effects of the drug. “They tend to focus on one-year outcomes but actually the boys that have been on the drug for longer periods of time are really showing tremendous benefits,” he says.

Even members of the Duchenne community whose sons are not among the 13 percent of sufferers the drug treats feel this is their strongest hope. Like Debra Miller, president of Cure Duchenne, whose only son Hawken 19, suffers from a different mutation of the disease.

“I’m pushing just as hard as all the other parents because we know that until these drugs are approved the companies will not be able to commit to a whole pipeline of other drugs for the rest of the boys,” Miller tells PEOPLE. “If this is not approved there is no other drug for the families to turn to.”

For now, all the families can do is wait – and hope that good news will come with the new year.

“I don’t have a lot of time right now,” Woods says. “My son is facing complete paralysis of his whole body, heart failure, pulmonary function problems – out of all of those things, what risk could there be that could outweigh the end result of the disease?”