For years, it was tradition across the country for families to tune into the Jerry Lewis MDA Labor Day Telethon. For most of its time on the air, it was broadcast on 3TV and hosted from our local studios. The telethon is no longer around, but the money raised during those years is still hard at work.
The Beurlein Family of Phoenix spent years participating in the telethon. Brothers Steven and Nick were diagnosed with Spinal Muscular Atrophy, a form of muscular dystrophy, at a very young age. The boys, along with their parents, would appear on TV and help raise funds in hopes of one-day finding treatments and maybe a cure for SMA.
Now ages 29 and 27, the Beurleins are about to try the first FDA-approved treatment for SMA.
“I’ve got decent strength. But as the years go on, it’s gotten harder and harder, just doing the simple things of cutting food, brushing teeth,” said Nick. “It just feels like a 2-hour workout somedays.”
Nick has lost more strength over the years than Steven, who is able to work part-time from a computer in their Valley home. But Nick hopes that he could also have a job if this new drug restores some of his strength.
Dr. Saunder Bernes of the MDA clinic at Phoenix Children's Hospital says the drug Spinraza has been very successful in many of the younger SMA patients being treated.
“It’s probably the biggest thing I’ve seen in my career treating neuromuscular children,” said Bernes.
The drug is helping some patients to regain the strength they’ve lost. For others, it’s preventing them from losing further strength. Some babies born with SMA and treated with Spinraza are actually learning to walk.
Dr. Bernes calls the drug game-changer and says it’s helped reclassified SMA as a “treatable disease.” Because of that, it’s expected to soon be added to newborn screening tests.
The Beurleins say they’re cautiously optimistic about Spinraza, as they know they’re older than some of the younger patients seeing remarkable success.
“It’s exciting to see if we get back to strength, and how much each of us might get,” said Nick. “Any good result is positive,” Steven said.
For more information on SMA, Spinraza or the MDA:
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