A Baby Girl With A Rare Disorder Needs A Special $2.1 Million Medication To Live Past 2

by Karen Belz
Karen Belz has written for sites such as Bustle, HelloGiggles, Romper, and So Yummy. She's the mom of a sassy toddler and drinks an alarming amount of Sugar-Free Red Bull in order to keep up with her.

There are a lot of unexpected situations that might happen after you have a child. For one, even though we’ve been procreating since the beginning of time, labor and delivery can still be risky.

Secondly, your baby might be born with an illness you never expected.

Ceri and Rory Devine live in California, and they’re the parents of a 6-month-old named Rhys. From the outside, Rhys looks like a happy baby. But she’s fighting a rare disease that many children who have it don’t overcome. When Rhys was only 4 months old, Ceri realized her baby was having difficulty lifting her head.

“She always struggled with tummy time, and wasn’t able to roll over or hold her head up for very long,” Ceri wrote on her family’s blog.

The physician they saw suggested physical therapy. However, things still weren’t improving.

“At her 4-month check-up, our pediatrician said she just had low muscle tone and recommended physical therapy,” she wrote. “At her next check-up at 6 months, the pediatrician immediately noted Rhys’ lack of progression and expressed concerns that she may have Spinal Muscular Atrophy, a genetic condition we had never heard of before.”

Hearing about a condition like this is a nightmare for parents. Ceri immediately went on Google and was devastated to learn more about the condition. Most babies who have it don’t live until the age of 2.

“We hoped it was something else less severe, easier to treat, less devastating. ANYTHING but SMA,” she wrote. “After 2 weeks, the longest 2 weeks of our lives, we received the heartbreaking diagnosis. Rhys has SMA Type 1, the most severe form of the disease.” SMA is a muscle degenerative disease. Muscle nerve cells are killed, which prevent a child from eating, drinking, and moving regularly.

Unfortunately, there’s no cure for the disease. But there are treatments in the works that make things much less devastating. One such treatment is called Spinraza, which first became approved for use in December 2016. But the problem is, Spinraza is incredibly expensive.

According to People magazine, Spinraza is typically administered every four months through a spinal injection. It reportedly costs $750,000 the first year and $375,000 every year after. Rhys would need to take this medication for the rest of her life, which is something that most families absolutely can’t afford.

And that puts Ceri and Rory in a really tough position. When it comes to the health of their child, there shouldn’t be such financial obstacles like this. A second drug named Zolgensma might also help her condition. However, that one happens to be even more expensive. Ceri writes that treatment with Zolgensma is $2.1 million. It’s known as the most expensive medication in the entire world.

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Happy 1/2 Birthday Sweet Stuff 🖤 #rhysdevine #6months

A post shared by Ceri Devine (@ceriouslydevine) on

“Because SMA Type 1 is aggressive and degenerative, this is a race against time,” writes Ceri. “With each day that passes, motor neurons in Rhys’ sweet little body are becoming inactive. In addition to the gene therapy, Rhys will need ongoing physical therapy and support equipment based on her needs to help her develop, improve mobility, and improve her quality of life.”

To try to raise both money and awareness, Rory and Ceri started Rise Up for Rhys. “We decided to start Rise Up for Rhys as a way for family, friends and others to offer support during this incredible difficult journey,” they said. “The name was inspired by Andra Day’s song, ‘Rise Up’, which tells a story of struggle and perseverance. In particular, it talks about moving mountains, which we have every intention of doing for Rhys.”

Keeping Rhys alive will be a lifelong mission, but it’s one that Rory and Ceri are both taking seriously. It seems as if during Ceri’s pregnancy, there was no way to tell that Rhys would have these complications. This life-changing diagnosis could have happened to anyone.

While raising kids is often expensive, nobody could have predicted these types of bills. Not only are Rory and Ceri learning more about SMA as time goes on, but they’re also open to sharing stories of other children who have similar stories. In every case, you can see how loved these children were during their fight.

According to SMA News Today, these new treatments will greatly help children with SMA. But they’re not exactly a cure — children may still have difficulties down the road. The publication wrote about a child with SMA type 0. The child was treated with both the aforementioned medications during her first year.

“The child continues to show motor improvements after one year of treatment, compared with how this disease would progress without treatment (its natural history), complications that include difficulties in breathing and swallowing, as well as heart and skin abnormalities, remain and affect her quality of life,” the article states. It’s a truly upsetting diagnosis to receive.

So how can you help Rhys? Financial donations are always appreciated, and her family set up a GoFundMe campaign to try to raise money toward lifesaving treatment. The GoFundMe page has been shared thousands of times, and they’re getting close to their starting goal. Any amount is bound to help.

So far, Rhys has reportedly gone on her first round of Spinraza — and it’s already made a big difference. “If you aren’t following along on Instagram, please do so you can check out Rhys kicking her legs more than we’ve ever seen before!” the family wrote on their GoFundMe page. “This is following her first dose of Spinraza, we were just amazed, which you can tell by our voices.”

“Thank you again, the Devine’s feel so loved and supported,” they continued. Sharing updates of Rhys is bound to be a great way to connect people to her story. Another way to support her and other children with her condition is to spread the word. Prior to the diagnosis, it seems like Rory and Cheri didn’t know too much about SMA. Now they do.

While diseases like this may be rare, they still exist — and they can make things very difficult for families. Hopefully, in time, these medications won’t be quite as expensive. But since time is so limited and SMA can be fatal, there’s no other option right now for families to try to save their children.