Hundreds of patients have restarted
SPINRAZA® (nusinersen) after discontinuing

NINA:

What up Brooklyn! How’s everybody doing tonight?

Being in a wheelchair is kind of like being a celebrity. When people stare at me in the street, I wave.

NINA:

I started getting into comedy; as far as acting, when I took a class and I met an amazing, amazing teacher, and she’s the one that actually who took me to a open mic night that I performed at for the first time.

NINA:

I never ever have to wait on line for anything. Come with me to a theme park some time.

NINA:

Yeah, I won $25. So after that, you know, that was it, I was hooked.

NINA:

My name is Nina, I’m 39, and when I was 18 months old, I was diagnosed with SMA Type 2.

NINA:

My childhood was great–I loved my childhood. I had a lot of fun.

Very early on, I had a sense that I was different.

This chair is a big part of me that nobody else really understands.

NINA:

When I was diagnosed, they had very little hope for me to even live past a teenager.

My condition of SMA? It was hard–it was very hard on me.

I started developing muscles weakening from the SMA.

I started getting very weak, my arms especially, and my neck. There were days where I couldn’t even pick my head up.

NINA:

So for a few years, I had been doing research, and that’s when I came across they were doing the trials for SPINRAZA.

VOICEOVER:

What is SPINRAZA?

SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.

VOICEOVER:

SELECTED IMPORTANT SAFETY INFORMATION:

A healthcare provider will monitor for signs of bleeding complications and kidney damage while taking SPINRAZA.

VOICEOVER:

These are not all the possible side effects.

Please continue watching for additional Important Safety Information

BACKGROUND MUSIC [AUDIENCE CLAPPING]

NINA:

SPINRAZA became available to me at like, just the right time, because I was at a point where I was starting to lose hope.

NINA:

The first thing that I noticed were the improvements in my sitting ability and how I was able to use my arms, which was clear when it came to my crafting.

This is my experience. Others may be different.

NINA:

I can fold up a piece of paper in, like fours, and rip it, and I’ve always had trouble doing that. So that was pretty awesome.

When I did my yearly evaluation with the Hammersmith Test, I am feeling like I’m gaining in certain aspects.

NINA:

VIP rooms, I’ve gotten into for free. I mean look at this room tonight. They’re paying me to be here.

VOICEOVER:

SPINRAZA, also known as nusinersen, is given as an injection into the lower back; a procedure called an intrathecal injection. Treatment with SPINRAZA starts with 4 injections in the first 2 months as initial doses, and after that SPINRAZA is given 3 times a year.

Some lab tests were run when first starting SPINRAZA and again before each dose to monitor their blood and kidneys as recommended by the doctor. The most common side effects of SPINRAZA include lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome.

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

NINA:

After being on SPINRAZA for about a year and a half, I decided to stop for a little while.

NINA:

So I’ve been back on SPINRAZA for about a year now.

I have a great Family Access Manager and she’s always on top of me, making sure I get to my appointments…she makes sure all the approvals go through.

She definitely has my back–absolutely.

NINA:

We always get the best parking spaces, that’s a given.

NINA:

I would say to people thinking of trying SPINRAZA to definitely call your doctor and talk with them about it, and see if it’s the right thing for you. It was definitely the right thing for me.

A motto in my family is “Never give up. Go under, go over, go through, but never give up.”

If I want to go do acting, I can go do acting. You know, if I wanted to go figure skater, I mean maybe not that but…

NINA:

I know it’s not a cure and my SMA can still progress, but I’m happy with my experience and what SPINRAZA has done for me.

NINA:

Thank you so much. You’ve been a great audience, love ya.

[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.]

Speaker: Voiceover

WHAT IS SPINRAZA?

SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.

SELECTED IMPORTANT SAFETY INFORMATION

A healthcare provider will monitor for signs of bleeding complications and kidney damage while taking SPINRAZA. These are not all the possible side effects.

Please continue watching for additional Important Safety Information.

BACKGROUND MUSIC

NASSIM RAD, MD:

Patients come back to SPINRAZA because of their own preferences and goals. I am currently treating 47 adult patients with SMA. I have patients who have started and stayed on SPINRAZA because it fits with their goals. Some of my patients have restarted SPINRAZA.

BACKGROUND MUSIC

CRYSTAL PROUD, MD:

The communication strategy often involves addressing patient concerns, emphasizing the importance of treatment, and fostering an informed decision-making process to ensure patients feel informed and involved in their care.

BACKGROUND MUSIC

NASSIM RAD, MD:

When patients are on SPINRAZA or when they restart SPINRAZA, we like to monitor for safety, as well as monitor motor function and range of motion, looking for either gains or losses of functional ability.²

CRYSTAL PROUD, MD:

It’s important to reestablish a baseline functional assessment, and I reassess my patients every 6 months so that we can discuss the impact of their treatment on their motor function.²

BACKGROUND MUSIC

VOICEOVER:

WHAT IS SPINRAZA?

SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.

IMPORTANT SAFETY INFORMATION

Increased risk of bleeding complications has been observed after administration of similar medicines. Your healthcare provider should perform blood tests before you start treatment with SPINRAZA and before each dose to monitor for signs of these risks. Seek medical attention if unexpected bleeding occurs.

Increased risk of kidney damage, including potentially fatal acute inflammation of the kidney, has been observed after administration of similar medicines. Your healthcare provider should perform urine testing before you start treatment with SPINRAZA and before each dose to monitor for signs of this risk.

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

Before taking SPINRAZA, tell your healthcare provider if you are pregnant or plan to become pregnant.

Please see full Prescribing Information via the link provided.

This information is not intended to replace discussions with your healthcare provider.