Real-world SPINRAZA stories

BACKGROUND MUSIC

MAYA: Hi, I’m Maya, I’m from Texas, and I’m a teen living with SMA.

BACKGROUND MUSIC

MAYA: Um, something that I would like to change people’s mindset is, the, um, SMA doesn’t define you as a person. Like, you’re more than just someone in a wheelchair, and that really means a lot to me. Because, whenever people like see you, they’re like, “oh yea, you’re in the wheelchair, I know you,” and like, no, and they see who you really are once you spend time with them. They’re like, “oh, you’re actually pretty cool,” and that’s really nice.

MAYA: I went to homecoming my freshman year with my friends, and they helped put my knee bar on and my top bar on to get me situated so I could stand up at homecoming and take pictures with my friends, and dance with them in my chair but standing, so that was really nice.

MAYA: My friends take care of me and I’m really appreciative of that. My mom doesn’t have to come everywhere with me and that’s really big.

[ISI present in video and available below.]

BACKGROUND MUSIC

MAYA: I started taking SPINRAZA in March of 2017, so I was 9, and I’ve taken it for over 6 years, and so I’m 15 now. It’s been a while…so that’s really nice.

Keep in mind, that this is my experience with SPINRAZA, others may be different.

MAYA: For my first initial doses, the first time I had really bad nausea, and uh, spinal headache, it made me throw up the first time. I had headaches throughout all those loading doses.

MAYA: SPINRAZA is given as an injection into the lower back, a procedure called intrathecal injection, delivering medication directly into the central nervous system. Treatment with SPINRAZA starts with 4 injections in the first 2 months as initial doses, and after that, SPINRAZA is given every 4 months.

Some lab tests are run when first starting SPINRAZA as recommended by medical professionals. They are run again before each dose to monitor their blood and kidneys. Talk to your doctor about risks and benefits before starting SPINRAZA.

BACKGROUND MUSIC

BACKGROUND MUSIC

MAYA: I remember being able to open markers, which I couldn’t do that before. So, my motor skills have gotten better. My strength has gotten better. I can lift 1-pound weights, like, I can curl those. I couldn’t do that before so that’s really big for me. I’m grateful for how far I’ve come.

BACKGROUND MUSIC

[ISI present in video and available below.]

JENNIFER (KIRRA’S MOTHER): You deliver this beautiful baby and you hold your baby and the first phone call is not mom, dad, brother, sister. Your first phone call is insurance and your doctors. And so it’s hard sometimes, but that’s what’s at the top of your list because you know that that’s going to get the ball rolling.

PEARL ANN (MAVRIK’S MOTHER): It’s very important to diagnose SMA early. The faster you know, the faster you can react.

ALYSSA (JUDE’S MOTHER): We have five children, and two of them have spinal muscular atrophy. Because his older sister has SMA, Jude was diagnosed in utero via amniocentesis.

JENNIFER (KIRRA’S MOTHER): We had lost a child, and we were living with Nathan, who was experiencing SMA daily. With Kirra’s diagnosis, we were heartbroken. You spend a few days crying. You spend a few days not knowing what to do next, and then you just know it. Mom takes over, and you just know that whatever it looks like, I’m going to do whatever I can for my baby, and I think that that’s any parent.

FEMALE VOICEOVER: In 2016, SPINRAZA® (nusinersen) was approved as a prescription medicine for infants, children, and adults living with SMA. A health care provider will monitor for signs of bleeding complications or kidney damage while taking SPINRAZA. These are not all the possible side effects.

These are stories of 3 families living with SMA. This is their experience. Others may have different experiences.

JENNIFER (KIRRA’S MOTHER): Kirra is my five-, almost six-year-old daughter. She is spunky and feisty, a sweet little personality, my little sidekick. Kirra likes to roller-skate, to play outside, to ride her bike, to go for walks. She loves to snuggle and read a book.

PEARL ANN (MAVRIK’S MOTHER): Mavrik is quite the character in his own little way, and he really loves dinosaurs and playing with his siblings.

ALYSSA (JUDE’S MOTHER): Jude likes to play outside and play with trains…play with his siblings. We read books. He likes to shoot rockets and throw balls. He’s just a quiet-natured kid. Knowing that SPINRAZA was available gave us a lot of hope.

JENNIFER (KIRRA’S MOTHER): I don’t even know if I can put into words how excited we were that our baby was going to be treated. And so, Kirra was dosed at 11 days old.

ALYSSA (JUDE’S MOTHER): We learned about SPINRAZA as a treatment because Annabel’s physicians told us that was an option. And so when Jude was born, we already knew that that was a treatment option for him. Jude started treatment before SMA symptoms started showing up.

PEARL ANN (MAVRIK’S MOTHER): The neurologist did recommend SPINRAZA and he received his first dose of SPINRAZA at seven weeks old. This is what Mavrik needs. This is what he gets.

A few of the milestones I’ve tracked in the past was rolling over, sitting, supported, unsupported, and learning to crawl.

Those were very tear-jerking milestones for us.

Milestones we’re currently working on is walking without assistance.

JENNIFER (KIRRA’S MOTHER): Kirra’s doing a great job meeting her milestones. She’s sitting, walking. She is a joy to watch.

ALYSSA (JUDE’S MOTHER): Jude did achieve motor milestones, just in a delayed pattern. So he was able to sit unassisted, but it was after the six-month mark. And he walked, but it was after he was a year old. He hasn’t started running yet, but I have hope that he will.

JENNIFER (KIRRA’S MOTHER): We didn’t know what to expect. So when Kirra Faith started cruising in our living room and holding onto our couch, and when you could hold her hands and walk across the room, there are no words.

SPINRAZA is given as an injection into the lower back, a procedure called an intrathecal injection, delivering medication directly into the central nervous system.

Treatment with SPINRAZA starts with four injections in the first two months as initial doses, and after that, SPINRAZA is given every four months.

Some lab tests are run when first starting SPINRAZA, and as recommended by a medical professional, they are run again before each dose to monitor their blood and kidneys.

Talk to your doctor about risks and benefits before starting SPINRAZA.

PEARL ANN (MAVRIK’S MOTHER): I want other parents to know they’re not alone.

ALYSSA (JUDE’S MOTHER): I think there’s a lot of hope.

JENNIFER (KIRRA’S MOTHER): You will get through this and you don’t believe that you can, but you will.

ALYSSA (JUDE’S MOTHER): SPINRAZA gave us hope and we want other families to know that it can give their children hope as well.

JENNIFER (KIRRA’S MOTHER): We talked to her doctor about SPINRAZA. We don’t see any reason to change. She’s doing amazing. She’s living with SMA and she’s living with SPINRAZA, and we have found a happy medium. She’s doing great, and we’re so grateful for what she is doing.

[ISI present in video and available below.]

FEMALE VOICEOVER: If you are considering treatment, talk to your child’s doctor about SPINRAZA.

KELLI (MOTHER OF CARLEE): To think of who Carlee is at 11 and see who she’s going to be when she’s 25 or 35 or 45— she’s going to change the world.

My name is Kelli, and my husband Jason and I have been married for 20 years. We have two beautiful kids, Drew, who is 16 years old, and Carlee, who is 11.

Carlee is a typical 11-year-old little girl, a tween, if you will, and she is pretty amazing. She has a lot of life in her, and she just, she radiates. She loves cheerleading. She loves dancing. Drew was a pretty typical kid, and he hit all kinds of milestones. And when Carlee did not, I just kind of figured something was going on. So I went to our pediatrician and basically was told she could be developmentally delayed. We went to the local children’s hospital for additional testing. April 19, 2010, totally changed our lives. To hear the words spinal muscular atrophy, the expectations for our lives just totally changed in that one moment. We really just sat down and just said we can’t allow this to just overtake us and be everything of every day. We just gotta live.

You just do the things you gotta do in your life. When I first heard about SPINRAZA, we started seeing that more and more research was being done and it was getting closer and closer to FDA approval. We were like, when it comes on the market, what does that look like? You know, how does Carlee get that? We just said, we’re just gonna make it happen. And for us just to get that medicine to her as quickly as possible, I knew I had to be really proactive.

Carlee received her first treatment on March 31, 2017. She’s had her four loading doses and has received nine maintenance doses. SPINRAZA is given as an injection into the lower back, a procedure called an intrathecal injection, delivering medication directly into the central nervous system. Carlee first started treatment with four injections in the first two months as initial doses, and now she goes back once every four months.

Some lab tests were run when Carlee first started SPINRAZA, and again before each dose to monitor her blood and kidneys as recommended by our doctor.

Some folks were like, oh, you know, she’s going to walk. And that wasn’t our expectation. We are hopeful for our treatment with SPINRAZA, and that’s what’s exciting, is to see her being able to do things that she normally would do.

It’s going to be neat to see the opportunities she’s going to create for herself. It’s not a cure; it’s a treatment. There’s a difference. And it’s a beautiful treatment for our daughter.

[ISI present in video and available below.]

ANTONIA (MOTHER OF ASHLEY): I have an amazing child, and SMA doesn’t define her.

She is all love and all happiness and she projects that into everybody.

Hi, my name is Antonia. I am a mother of a wonderful seven-year-old girl by the name of Ashley. She is diagnosed with spinal muscular atrophy Type 3.

Ashley is an amazing little girl. She is curious about the world. She wants to know everything.

She’s very loving, very kind. She was diagnosed back in July of 2015. You know, I didn’t know what was wrong with her. I went into the hospital to the doctor with a mindset that whatever it is, we’re gonna tackle it, we’re gonna be okay. And then the following words were, your daughter will probably not make it past 10 years old. And it’s like, my whole world crumbled. How do you tell that to a parent? So when I heard that SPINRAZA was in the market, that the FDA approved it, it’s like, that’s my hope. That’s what I’m gonna hang on to.

Now that Ashley is seven years old, she is open about her medical condition. She says, oh, I’m a little sore. I get tired and I need to take breaks. She’s like, but that’s okay, I can play other things.

Her big thing is, I wanna be a scientist, great. Mommy, but I also wanna sing and I also wanna go to the moon. I’m like, well, you can be everything. I really just hope for her to maintain the strength that she had. Ashley had her first dose of SPINRAZA in July 2017.

SPINRAZA® (nusinersen) is given as an injection into the lower back, a procedure called an intrathecal injection, delivering medication directly to the central nervous system. Ashley first started treatment with four injections in the first two months as initial doses, and now she goes back once every four months.

Some lab tests were run when Ashley first started SPINRAZA, and again before each dose to monitor her blood and kidneys as recommended by her doctor.

I’ve just noticed that on her Hammersmith, she’s gained, I think she’s up from her initial testing. Her six-minute testing where she walks, she’s gained a couple meters. I really admire that she tries to be as active as possible. She can’t run as fast as other kids, but she sure does try. I want her to have the best chance of life that she should have, that she deserves to have. Please remember that this has just been our experience, and yours may be different.

I really admire that Ashley can speak for herself, and she’s very outgoing. She doesn’t let things get her down. She’s everything that’s positive from her joy to her feeling that SMA is not a limitation. I think it’s important that Ashley sees that regardless of anything going on you can still accomplish so many things. The little gains means so much. SPINRAZA has given us that.

[ISI present in video and available below.]

GINA (RUBY’S MOTHER): I have three little girls who are busy and full of life. I have a 10-year-old, a 7-year-old, and a 4-year-old. They are precocious and fun. Ruby is our youngest daughter. She has Type 2 SMA. She’s four years old. She is full of life.

She’s a super joyful, playful, happy kid. She is like the sun, I think, in our world. She loves to be kind of a big kid and to get to do what her sisters are doing.

STEVE (FATHER OF RUBY): Emma is our middle daughter. She’s seven years old now. She at age three began to show some signs of abnormal walking, so she was considered a Type 3 child at the time of her diagnosis. After the diagnosis, we were scouring. We came across SPINRAZA.

FEMALE VOICEOVER: SPINRAZA is given as an injection into the lower back, a procedure called intrathecal injection, delivering medication directly into the central nervous system. SPINRAZA starts with four injections in the first two months as loading doses, and then once every four months as maintenance doses. Some lab tests were run before Emma and Ruby first started SPINRAZA, and again before each dose to monitor their blood and kidneys as recommended by their doctors.

STEVE (FATHER OF RUBY): The care team is really there to help support us through the process. However, our kids and us as parents are still in the driver’s seat. I think as a parent or person, you just adapt. And I think our daughters have adapted.

GINA (RUBY’S MOTHER): For Ruby, I can give her markers, paint. She can pick a paintbrush up and, you know, push it across the paper and something shows.

STEVE (FATHER OF RUBY): Ruby is so excited and proud when she goes to preschool now, and she can be with her classmates and create a picture.

GINA (RUBY’S MOTHER): Her preschool teacher actually tells us that she is the one who completes the art projects more than any of the other kids, and she’s so proud of them. So each one of those minor movements has been monumental.

She’s amazing. It’s amazing, and it is breathtaking every time.

STEVE (FATHER OF RUBY): In the entire history of human existence, there’s never been a drug for this disease. And now there is. We were facing some really big and hard challenges with our girls, and we realized that those things may still happen over time, but we feel like we have this tool to fight back with.

GINA (RUBY’S MOTHER): They are living their lives. They’re learning joy. There’s magic in their world. And I’m really proud of us that SMA is not first in our world. Their childhood is. Their joy, creativity, and learning to love the world around them and to see everything and to find the beauty in all of it.

[ISI present in video and available below.]

RUBY’S SISTERS: How do we turn this video off?

INTERVIEWER: I think it’s done.

RUBY’S SISTERS: It is?

INTERVIEWER: Yeah I think so, right?

RUBY’S SISTERS: Bye! Hakuna Matata!

SOFIA’S SISTER: My sister Sofia is goofy, and she is also silly.

JASON (SOFIA’S FATHER): Sofia is just the most amazing little girl. She has got so much joy and so much love and so much drive. She really loves ballet, she loves dancing, she loves singing. When we’re in the car, you know, she’s always asking us to play certain songs and sing. It’s so cute to listen to.

NATALIA (SOFIA’S MOTHER): She’s a demanding little girl. Everything is hers. She has to try everything. She wants to do everything, be included in everything. Whether she can or can’t do something, she’s going to try.

The doctor had told me that she tested positive for spinal muscular atrophy. They pretty much just told me, enjoy the time that you have with her.

JASON (SOFIA’S FATHER): You feel like you don’t have hope. You feel like there’s nothing out there. That’s what they tell you.

NATALIA (SOFIA’S MOTHER): SPINRAZA is given as an injection into the lower back, a procedure called intrathecal injection, delivering medication directly to the central nervous system. Sofia first started treatment with four injections in the first two months of loading doses, and now we go back once every four months for her maintenance dose. Some lab tests were run before she first started SPINRAZA, and then again after each dose to monitor her blood and kidneys as recommended by her doctor.

After we began the treatment, it took a few months before we started to see some changes. She was starting to be able to hold her head up independently without us having to use our hands to help her. And then eventually her legs started to have more movement. She was able to start lifting one leg in the beginning. And then with lots of physical therapy and practice and repetition, she started being able to lift both legs up.

JASON (SOFIA’S FATHER): Since she started ballet, it’s been really good because now she’s been able to be around other kids her age and just feel like she’s part of something. It’s just a joy to see her buzz around in her little walker and be able to walk around with the girls who are dancing and put her hands up in the air and pose and bow. She loves being outside. She really loves doing anything physical that she can. She’s so driven. I’ve never seen such a drive in anybody. I mean, even though she knows she’s held back from her disease, she’s so focused and driven on what she wants to do.

NATALIA (SOFIA’S MOTHER): We’re looking into adaptive skiing for Sofia because she loves being outdoors. She loves the snow.

JASON (SOFIA’S FATHER): She’s such a strong fighter. And to see how resilient a little girl like that is with this disease and just keeps moving forward and fighting is just an absolute gift. We’re so blessed.

[ISI present in video and available below.]