The SMA Miracle: First Ever Treatment Approved for Rare Disease

Imagine walking through the woods at night. You have no lantern and no flashlight—not even a match. The darkness is suffocating, and you’re terrified. But you keep blindly stumbling.

It’s like life without hope. You yearn for light, yet you learn to live without it.

That’s what young Sara and her parentsBobbi and Brian, had to learn since her diagnosis with spinal muscular atrophy (SMA), a debilitating disease with no cure.

What’s it like with SMA?

Sara moves with help from an electric wheelchair and requires a feeding tube. SMA weakens her to the point that she can’t wrap her small fingers around a pencil. Often, it’s even hard to breathe.

For Sara, this is life. And it’s a life she and her parents learned to live without hope for treatment.

Not anymore.

The FDA recently announced the first-ever approved therapy for SMA: SPINRAZA.

Imagine then, the teary-eyed, joy-filled reaction of Bobbi and Brian to what the Daily Republic describes as nothing short of a “miracle.”

Imagine again, that endless walk through woods swallowed in darkness. Except, now, in the distance, the sun starts rising, painting the darkness with purple, blue, and orange.

Hope. Source:

In a message to her friends, Bobbi wrote:

“Just to see Sara be able to hold her head up. Write with a pencil. Eat and swallow food. Stay healthy and stay alive!”

And it’s a reaction likely echoed around the communities and families of those diagnosed with SMA, because as Cure SMA states:

  1. The disease affects around 1 in 10,000 babies
  2. It’s the number one genetic cause of death among infants

If you’re part of this community, I encourage you to find out more about Spinraza here. Tune in to the webinar coming up on Tuesday, Feb. 14 to learn more about access to SPINRAZA.

Even if you’re not, I think Sara’s story is a needed reminder that hope springs up even at the most unexpected times, and when it does, it turns the darkness into something beautiful.

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