Family fights, hopes for cure for daughter’s spinal muscular atrophy before it’s too late
Gwendolyn Strong reached a major milestone when she turned 2 in October: she joined the rare 10 percent of those afflicted with spinal muscular atrophy who live past their second birthday.
When she was born to Bill and Victoria Strong in 2007, she was a normal, healthy girl who unknowingly had SMA, a degenerative disease which manifests itself by an inability to produce protein for muscles, which in turn affects the entire body.
By the time she was 6 months old, the symptoms had begun to manifest themselves. Her parents were told there is no treatment for it, no cure for their daughter, and to go home and enjoy what little time they had.
"For us, doing nothing was not an option," Mr. Strong explained. "We wanted to go home and fight this disease, along with Gwendolyn."
The Gwendolyn Strong Foundation was formed with two goals in mind. The first was to increase awareness of the disease, which while it remains the largest genetic killer of infants, is still largely unknown. Although it affects between 5,000 to 6,000 children at birth, very few survive to become living examples of the disease. Half of them do not make it past their first birthday.
The second goal is fundraising for research, and the foundation recently received a big boost. A Facebook vote ranked the foundation as one of the top 100 charities in the country, and the foundation received $25,000 from JP Morgan Chase.
The next Facebook vote, in January, gives them a chance to win $1 million.
If they win, the money would be divided up between similar projects. The $25,000 went to a research project at UC Irvine, where researching are conducting work that Mr. Strong called "a game changer."
"It's amazing when you see the research first-hand, and what they're doing with animals," he said. "They are very close to curing this disease, and they're doing that with $2 million."
While Spinal Muscular Atrophy may not be one of the most well- known diseases, it does offer a high probability of being cured soon. Projects at Stanford, UCLA, Cedars-Sinai and elsewhere are attacking the disease from different angles, trying to find a workable cure. One project involves triggering a gene to begin producing protein again, another implants nodes in the spinal cord, but Mr. Strong said the consensus in the scientific community is that it will be cured within his lifetime. In testimony before Congress, 50 of the leading scientists said that with proper funding, the disease could be cured within five years.
"When you're sent home with so little hope, the truth couldn't be further from that," Mr. Strong said. "I know our situation is challenging, but at least we have that to hold on to."
Finding a cure for SMA would only be the beginning of what that research could accomplish. Because the research for the disease is so promising, many scientists view it as a "gateway disease" that can find new options for other degenerative genetic diseases, such as Parkinson's, Alzheimer's or Lou Gehrig's diseases.
In the meantime, however, the Strongs continue to take care of Gwendolyn as best they can. Even though she has spent a total of three months of her life in the hospital, she is one of the healthiest babies afflicted with SMA. The disease affects how she can fight other illnesses (she cannot cough, for example) and her parents describe themselves as germophobes, doing everything they can to avoid contamination.
Gwendolyn has not yet lost the ability to move her hand, which would allow her to use a motorized chair if the spread of the disease can be stopped in time, or smile at her parents. However, there is a moment when SMA victims will become "locked in," and they lose all ability to control their bodies.
"We definitely did not begin with the false hope that we could save her," said Mrs. Strong. "But sometimes it feels like a race. When you see all this research and what they are doing, you can't help imagining what this can do. I mean, we're parents."
Those who wish to vote for the $1 million prize can register at causes.com/gsf, or visit the foundation Web site at gwendolynstrongfoundation.org. The voting is free and will take place from Jan. 15 to Jan. 22, an in addition to the top prize, there will also be five additional prizes of $100,000 each.
"Everyone in the community can help make this happen, not only for SMA but for so many diseases," Mrs. Strong said. "It's not every day you get to be part of a miracle."
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