Real-world SPINRAZA stories

STEPHEN: That’s fine if you want to look at me like an inspiration, but really the goal is to be more of an example.

My name is Steve. I’m 39 years old. I have SMA Type 2.

I live with my wife Brittany. We have been married for almost 9 years. I work full time with veterans. I’m also a foster parent. Lots of things going on.

I have a son who’s 19. He’s off at college. And my daughter is a typical 12-year-old sixth grader. You know, when you have a kid who’s just, for whatever reason, struggling because of what they’re dealing with, it’s just that reminder, you know, like, yeah, I understand this is going on, but if I can do it, you can do it. And so I think that’s really important to show the kids that they can basically do anything that they set out to do.

And so I think having SMA is actually an advantage to being a parent. Believe it or not, I do enjoy going to work, you know, I have the opportunity to work from home also, but I do prefer going into the office. I would like to think that my colleagues don’t see me as an inspiration, but as an example. It wasn’t until SPINRAZA came onto the scene that I started to kind of reintegrate, finding a neurologist. I thought it was important to pursue treatments. Ultimately, I was able to find a doctor who was treating adults with SPINRAZA. We agreed that SPINRAZA was a good choice for me. I sent my start form in to Biogen, and that’s when, a couple weeks later, I was assigned a Family Access Manager. I remember early on with all the questions as far as finding a treatment site, any kind of insurance stuff. She had a team that would help with that. She has been with me throughout this entire process.

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

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

I used the adaptive equipment to type, so that’s a lot of hand and arm movement.

My expectations for SPINRAZA were quite simple. I didn’t have any expectations. So I was hoping just to maintain having SMA. I set the example by doing things that could be perceived as difficult.

Yeah, I still have SMA, but I don’t let SMA stop me.

[ISI present in video and available below.]

YARIANNA: Here, tucked away, hidden. No one can see what matters to me. This is not that kind of story. No.

My name is Yarianna. I have SMA Type 2. I’ve been doing art my whole life. I also teach. It’s who I am. I didn’t want to stop doing any of it. But my SMA had me concerned about keeping up with my art and teaching. My first day of teaching, I went home crying. I was discouraged.

By the end of the day, I was struggling to lift my hands to the keyboard to advance my slide presentation. I’ve always wanted the world to see my art, what these arms, these fingers can do. I’ve always had the desire to express myself. Of course, there’s going to be challenges. But more than anything, I wanted to maintain my muscle strength to continue painting and teaching. My doctor suggested SPINRAZA, and I was ready.

SPINRAZA is started with four intrathecal injections in the first two months as loading doses, followed by once every four months for maintenance dose.

I experienced improvements. My physical therapist saw it. She performs tests like the revised upper limb module.

It shows how well I can do things like trace a simple maze with a pencil or raise my hands above my head. Treatment with SPINRAZA has helped. You see how I’ve continued with my art? This was my experience with SPINRAZA. Not all patients experience improvement. I’m excited to know that there’s a treatment that may help stabilize or even improve function for adults with SMA.

Talk to your doctor about the possible risks and benefits before starting SPINRAZA. Some lab tests are run before starting SPINRAZA to monitor blood and kidneys as recommended by your doctor. And you will continue to have ongoing lab tests before each dose.

My doctor told me the most common side effects of SPINRAZA include lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome.

And these days, I can lift my brush to the canvas, and I can paint, and I continue to teach. Oh, I love to see my students’ first reactions. My blue hair, these tattoos, the wheelchair. My students have always appreciated me for who I am. They see me paint, hear me speak. I leave quite an impression. At this point, I have created enough work as an artist to have a showing one day. That’s my next goal. I know who I am. Teacher. Rebel. Artist.

[ISI present in video and available below.]

RYAN: Hi, my name is Ryan. I’m 21 years old. I’m a fourth year at college, and I live with spinal muscular atrophy Type 2.

I started treatment on SPINRAZA when I was in high school, a senior in high school.

I decided on transferring to a university after two years of living at home. I was really eager to kind of get out and live life and see the world. My parents told me that, Ryan, you can do anything. You’re away at college. You can make the most of your two or three years there. So they were a really big support system for me and one I leaned on for guidance. I’ve been living on my own with the help of caregivers. I have a solid caregiver schedule. They’re all great friends, too. So I’ve been doing it for about a year, planning my daily schedule. When do I have caregivers do laundry? When do I cook dinner and lunch? Do all these things that I usually haven’t had to manage in the past. Going to class every single day is kind of the routine that I have. And I’ll travel to campus and go to classes in my wheelchair and be with friends in class and then I’ll maybe grab a bite to eat.

I actually got involved in our student government. A lot of people came to me and they said, Ryan you would be really great if you ran for president. And I said, what? Like, no way. I ran and got elected so I serve as president and got really heavily involved in terms of disability rights and transfer advocacy as a transfer student.

Before I decided on changing treatments, I was on SPINRAZA for about three and a half years. Once I got on the new treatment, I told my doctor exactly what was happening. She was really understanding. She said, Ryan, listen, we can get you switched back.

I was starting to notice at the time some things that I could do after SPINRAZA, there’s this story that I share about going out to Mexican food with my mom. I kind of got a sense of confidence at that meal. I went to reach for each taco, and I was able to grip the taco and eat it and my mom, her jaw just dropped to the floor. She’s like, did you really just do that? I said yeah absolutely I did, and I was really proud of myself.

FEMALE VOICEOVER: Talk to your doctor about the potential risks and benefits before starting SPINRAZA. SPINRAZA is given as an injection into the lower back through a procedure called intrathecal injection, delivering medication directly into the central nervous system. Treatment on SPINRAZA begins with four injections in the first two months as loading doses, followed by a maintenance dose every four months thereafter.

Before starting treatment on SPINRAZA, and before each dose, lab tests are required to monitor for risk of bleeding and kidney damage.

RYAN: You know, I have a lot on my plate these days. I am a full-time college student. I’m involved in student government, working 19 and a half hours a week, maybe more. And I also have a very active social life in terms of just hanging out with friends and going to dinners. You know, of course I leave a little bit of room for studying, too. I realize that a lot of people’s experiences are different, so I’m grateful to be on a treatment that works for me. And for me, that’s SPINRAZA.

[ISI present in video and available below.]

LAUREN: I’m Lauren. I’m from Kansas, and I’m an adult living with SMA.

I was diagnosed with SMA when I was between two or three.

My parents thought something was wrong with me when I wasn’t hitting the milestones and I was falling down a lot. I had a muscle biopsy done on my leg and that’s when they told my parents that I had SMA.

Growing up and going to school with SMA was different, but it was I think a good different. My muscles aren’t as strong as everyone was the way I would describe SMA to my friends, but it’s definitely challenging because sometimes they’ll see me get up out of my chair and they’re not sure like why she’s using a wheelchair. I started SPINRAZA because my doctors and my parents were concerned that I wasn’t going to be able to walk for much longer and that my strength was declining. I discussed my goals through the process with my parents and my doctor, and they too thought that the goals of the treatment were for me to be able to keep walking and maintain my strength.

SPINRAZA is given as an injection into the lower back, a procedure called intrathecal injection, delivering medication directly into the central nervous system. I first started treatment with four injections in the first two months as loading doses. And now I go back once every four months for my maintenance dose.

Some lab tests were run before I first started SPINRAZA. And again, after each dose to monitor my blood and kidneys as recommended by my doctor.

I was anxious about SPINRAZA because I knew that a needle was going into my back, but at the same time I was very hopeful. More than anything, I just wanted to maintain my strength and be independent. My friend Faith and I really love to travel together. I’m really thankful for her and being able to help me whenever I need help. I love traveling because I get to see different sites, different experiences, try new things, and it’s been a huge part of who I am and who I’ve become.

Some of the places I’ve traveled to are Australia, Spain, and France. I think there’s always challenges when going to a different country or even a different state. When I was in Greece, I couldn’t really use my wheelchair to do anything and I had to rely on my friends to help me, and I was really fortunate enough to be able to travel with ten people who were willing to accommodate me and like carry me on their backs just to get me up to our villa.

I want to be independent as much as I can be. Brushing my hair, doing my makeup, being able to change my clothes on my own, being able to make dinner on my own. But more than anything, it’s just the maintenance of my strength. I’ve been exercising and going to PT, which has really supported my treatment with SPINRAZA. I was realistic about SPINRAZA and what it was going to do for me.

I want people to know that they can achieve things. With SPINRAZA, my journey continues to unfold.

[ISI present in video and available below.]

IAN: So there was a huge sense of urgency for me. I thought, I need to do this soon because if I decline further, I’m going to be starting at a lower point on the ladder so to speak. Hi, I’m Ian. I’m 36, and I have SMA Type 3. From the period of like 26 to probably age 31 I was just, you know, swimming, I was doing the treadmill, whatever, and I felt like the SMA was stable at that point, and I was just…I was moving around a lot and I felt really good. At age 31, I’m not sure really what prompted the decline. I understood that the disease is progressive and so I think that was just, it was just time for it to do that. And then around the age of, like right when I turned 33, I hit another drop and I really noticed that when I was like showering, it was really hard to like get my arms up in certain positions and things of that nature. And I was like, well, maybe this is just attributed to, I’m just going through some adjustments, and I have to work harder and do more exercises. So I did that, but it didn’t change. I pretty much resigned myself to, I was going to enjoy myself and live in the moment, and then when things get worse, I’ll deal with it. This first conversation with SPINRAZA, it was really positive and my neurologist didn’t even hesitate to say that this could be an option for me.

I asked her at that time, too, what was my time frame probably that I’d still be able to walk?

And she said maybe 10 years and then I’d probably not be walking and SPINRAZA would help with that. And so I was like, yeah, sign me up.

SPINRAZA is given as an injection to the lower back, a procedure called intrathecal injection, delivering medication directly into the central nervous system. I first started treatment with four injections in the first two months as loading doses. And now I go back once every four months for my maintenance dose.

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

Getting up from a seated position, that seems to be a little bit easier, like getting out of my car, getting up off of my sofa, seems to be a little bit easier. And definitely with the showering and stuff, it seems easier to move my arms higher and things like that. One of the nurses, he’s an awesome guy named Lester, really positive and very supportive. He was like, you’re walking a lot smoother, man. So people have seen really cool differences, which is, like, mind-blowing. I know that it can be really hard to take the first step for a lot of people, and I would just like to show people that you can take the first step and the steps thereafter.

A lot of times I think people don’t know how to go about doing that. I just want to show people at least my way that I did it.

[ISI present in video and available below.]

JONNA: One foot in front of the other. That first step is what matters most. What comes next may not always be simple. But neither is my story. Challenges may come, but I am determined that today, they won’t stop me.

My name is Jonna. I have later-onset SMA.

Growing up, I wasn’t able to run like other kids. And as an adult, walking gradually became more difficult. I couldn’t ride a bike alone, but I was able to use a tandem bike. At this point in my life, I may not have been walking on my own. I was furniture walking, stumbling through my house. I talked to my doctor, and I was ready for SPINRAZA.

SPINRAZA is started with 4 intrathecal injections in the first 2 months as loading doses, followed by once every 4 months for maintenance doses.

Since starting SPINRAZA, I’ve improved my ability to stand unsupported, which is part of the Hammersmith Functional Motor Scale-Expanded test that I have done with my physical therapist.

And these legs? They take me even further than before. I’m excited to know that there is a treatment that may help stabilize or even improve function for adults with SMA.

My doctor told me the most common side effects of SPINRAZA include lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome.

This is my experience with SPINRAZA. Not all patients experience improvement.

Talk to your doctor about the possible risks and benefits before starting SPINRAZA. Some lab tests are run before starting SPINRAZA to monitor blood and kidneys as recommended by your doctor, and you will continue to have ongoing lab tests before each dose.

Outside is where I love to be. Since starting SPINRAZA, I’ve experienced improvements in walking and, so far, I’ve maintained them. I want to maintain my ability to ride my tandem bike with my husband. Stairs still test me, but if I take my time, I can use them, which I continue to need to do as a court advocate for kids. While my SMA can be challenging, I still strive to do as much as I can each day. I am a wife, mother, nature lover.

[ISI present in video and available below.]

ROBB: It was Christmas Eve, and it felt like the best Christmas present I had ever got.

Hi, I’m Robb. I’m 37 years old. I live just north of Columbus, Ohio, and I have SMA Type 2.

I just enjoy being with family, being outdoors, and just enjoying everything. Currently, I’m teaching American history. I really, really enjoy my career. I try to bring some of my passion for education into the SMA community. I really enjoy it with the guys and gals that are, you know, transitioning from child air quotes to adulthood because you know it’s…it was a very uncomfortable transition for myself at that time. There wasn’t very many people I could look to that had gone through what I’d gone through. I was the only kid in a wheelchair, I’ll tell you that.

And I had friends that really supported me and a lot of that I owe to my parents and my sister because they…they didn’t make my disease this all-consuming part of everyday life. It just was what it was. I hadn’t thought about treatment in years until that Christmas Eve. I got a text from my sister, “Have you heard about SPINRAZA?” And she goes, it’s approved, it’s approved. It was the moment you’d been waiting for but you forgot that you were waiting for it. There’s no no moment that’s gonna come like that moment came because you didn’t see it coming, and it came and it was like, all right, this is the moment you’ve been waiting for. Let’s do it.

We have friends and family that ask me, how you doing, how you doing, how you doing? And there’s always a couple of things I go to. And one of those would be before I began treatment, you know, you get this piece of paper and it’s a little maze and you have to hold a pencil and complete the maze. Before treatment, I was not even able to hold the pencil. I’m now able to hold that pencil. I was able to draw on the maze just a little bit, maybe just an inch. And every time I do it, I get a little bit further. A little bit further. A little bit further. It motivates me and it’s that reassurance that, you know what? This was the right choice.

This is my experience. Other people’s experience may vary.

FEMALE VOICEOVER: Talk to your doctor about the potential risks and benefits before starting SPINRAZA. SPINRAZA is given as an injection into the lower back through a procedure called intrathecal injection, delivering medication directly into the central nervous system. Treatment on SPINRAZA begins with four injections in the first two months as loading doses, followed by a maintenance dose every four months thereafter.

Before starting treatment on SPINRAZA and before each dose, lab tests are required to monitor for risk of bleeding and kidney damage.

ROBB: Spending time with my granddaughter has been amazing. You know, just having the opportunity to, you know, do those grandfather things, let her sit on my lap and read her a book or watch a goofy cartoon or sit outside and fish or just, you know, sit down and relax and spend time with each other. Those are amazing moments, and I love every second of it.

[ISI present in video and available below.]

NICOLE: I am an overachiever, so I’m always, always going to strive to be better.

Hi, my name is Nicole, and I am living with SMA Type 3.

I’ve been told all my life, you know, there’s nothing we can do for you. SPINRAZA was one of the first pieces of hope. I knew that no matter what I was going to try it. Every time I talk about it it’s like I get chills. To tell you how serious I was about getting on SPINRAZA, it got approved on December 23rd. I was at my doctor’s office on December 26th.

This is my experience. If somebody asks me, oh you know I’m on the fence, I’m not sure. All I say is, do you want to try to maintain what you have? And I am just blessed with doing a job that I truly love. I live, eat, and breathe graphic design and creativity. I am guilty of being a workaholic.

I have an 11-year-old wonderful daughter and awesome husband. For me, it’s always been mind over matter, focusing on what you can do and working around what you can’t. When we go out, it’s not just hop in the car and go. No, it’s get the harness, get the leash, get the bag, get the sweater, get the this, get the treats, get the dinner. A lot of times when we go out, people don’t know what to make of us.

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

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

The first SPINRAZA injection, it was very stressful. There was a little bit of a fear of the unknown, but overall it was an amazing experience. Everybody has different experiences, and I am happy that I made that decision to get on SPINRAZA and continue treatment. On injection day, there’s always a little bit of anxiety, but all in all, it is always an amazing experience of gratitude because I am able to get it and I’m very thankful for it.

[ISI present in video and available below.]