Some individuals featured may be participating in Biogen clinical trials.
MAYA // AGE 15 // OUTGOING ADVOCATE
TREATED WITH SPINRAZA FOR 6+ YEARS
“SMA doesn't define you as a person. You're more than just someone in a wheelchair.”
BACKGROUND MUSIC
Super: MORE STORIES OF HOPE
Maya, a teen with spinal muscular atrophy (SMA), shares her treatment journey
Sponsored by Biogen
MAYA: Hi, I’m Maya, I’m from Texas, and I’m a teen living with SMA.
Super: MAYA
An outgoing National Honor Society student and SMA advocate
Maya is a paid spokesperson for Biogen.
BACKGROUND MUSIC
Super: MORE STORIES OF HOPE
Maya, what inspires you to share your story?
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.
Super: “SMA doesn’t define you as a person”
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
Super: MORE STORIES OF HOPE
What has your experience been with SPINRAZA® (nusinersen)?
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: In clinical studies, the most common side effects of SPINRAZA included lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome (headache related to the intrathecal procedure).
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
Super: The CHERISH clinical trial studied 126 nonambulatory individuals aged 2 to 9 years with later-onset SMA.
At 15 months, the average score for people taking SPINRAZA showed a 3.9-point improvement in overall motor function, as measured by Hammersmith Functional Motor Scale-Expanded (HFMSE) versus a 1.0-point decline in the untreated control group.
BACKGROUND MUSIC
Super: “My motor skills have gotten better.”
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
Super: If you are considering treatment, talk to your doctor about SPINRAZA
SPINRAZA.com
[ISI present in video and available below.]
Super: ©2024 Biogen. All rights reserved. 04/24 SPZ-US-5747 225 Binney Street, Cambridge, MA 02142
BACKGROUND MUSIC
Super: MÁS HISTORIAS DE ESPERANZA
Maya, una adolescente con atrofia muscular espinal (AME), comparte su experiencia con el tratamiento
Patrocinado por Biogen
MAYA: Hola, soy Maya. Nací en Texas y soy una adolescente con AME.
Super: MAYA
Estudiante extrovertida de National Honor Society y defensora de la AME
Maya es portavoz remunerada de Biogen.
BACKGROUND MUSIC
Super: MÁS HISTORIAS DE ESPERANZA
Maya, ¿qué te motiva a compartir tu historia?
MAYA: Lo que me gustaría… que cambie en la mentalidad de la gente es… que la AME no te define como persona. Eres más que alguien en una silla de ruedas. Eso significa mucho para mí. Porque cada vez que la gente te ve, te dice: “Ah, sí, estás en silla de ruedas, te conozco”. Y no. Luego ven quién eres realmente cuando pasas tiempo con ellos y te dicen: “Eres realmente genial” Y es muy bonito
Super: La AME no te define comopersona. Eres más que solo una persona en un silla de ruedas.”
MAYA: Fui a la fiesta de bienvenida, en primer año, con mis amigos y ellos me ayudaron a colocarme el equipo en las rodillas y la parte superior. Me ubicaron para que pudiera levantarme y hacerme fotos con mis amigos y bailar con ellos... en mi silla, pero de pie, así que fue muy bonito.
MAYA: Mis amigos me cuidan y lo valoro mucho. Mi mamá no tiene que acompañarme a todas partes, salvo que sea algo muy difícil.
[ISI present in video and available below]
BACKGROUND MUSIC
Super: MÁS HISTORIAS DE ESPERANZA
¿Cómo ha sido tu experiencia con SPINRAZA® (nusinersen)?
MAYA: Empecé a usar SPINRAZA en marzo de 2017, cuando tenía nueve años. Lo estoy usando hace seis años. Ya tengo quince años, así que hace tiempo ya; y está muy bien.
Ten en cuenta que esta es mi experiencia con SPINRAZA. Para otras personas puede ser diferente.
Super: Los resultados individuales pueden variar en función de varios factores, como la gravedad de la enfermedad, el inicio del tratamiento y la duración de la terapia.
MAYA: En mis dosis iniciales, la primera vez, tuve un dolor de cabeza muy intenso, que me hizo vomitar la primera vez. Tuve dolores de cabeza durante todas esas dosis iniciales.
Super: En los estudios clínicos, los efectos secundarios más frecuentes de SPINRAZA incluyeron infección de las vías respiratorias bajas, fiebre, estreñimiento, dolor de cabeza, vómitos, dolor de espaida y syndrome posterior a la punción lumbar (dolor de cabeza relacionado con el procedimiento intratecal).
MAYA: SPINRAZA se aplica en una inyección en la parte baja de la espalda (inyección intratecal) y el medicamento se administra directamente en el sistema nervioso central. El tratamiento con SPINRAZA empieza con cuatro inyecciones en los primeros meses como dosis iniciales y luego se administra cada cuatro meses.
La primera vez que se usa SPINRAZA, se hacen análisis, según lo recomendado por un medico. Estos se repetirán antes de cada dosis para vigilar la sangre y los riñones. Consulte los riesgos y beneficios con su médico antes de empezar SPINRAZA.
BACKGROUND MUSIC
Super: El ensayo clínico CHERISH estudió a 126 personas no ambulatorias de 2 a 9 años con AME de aparición tardía.
A los 15 meses, la puntuación promedio para las personas que recibían SPINRAZA mostró una mejora de 3.9 puntos en la función motora general, medida por la Escala Motora Funcional Ampliada de Hammersmith (HFMSE), frente a una disminución de 1.0 puntos en el grupo de control no tratado.
BACKGROUND MUSIC
Super: “Mis habilidades motoras mejoraron”.
MAYA: Recuerdo que podía destapar los marcadores, algo que antes no podía hacer. Mis habilidades motoras mejoraron. Mi fuerza mejoró. Puedo levantar pesas de una libra, ya puedo hacerlo. No podía hacer eso antes, así que es muy importante para mí. Estoy agradecida de lo lejos que llegué.
BACKGROUND MUSIC
Super: Si considera este tratamiento, hable con su médico sobre SPINRAZA.
SPINRAZA.com
[ISI present in video and available below]
Super: ©2024 Biogen. Todos los derechos reservados. 05/24 SPZ-US-5846 225 Binney Street, Cambridge, MA 02142
MAYA // AGE 15 // OUTGOING ADVOCATE
TREATED WITH SPINRAZA FOR 6+ YEARS
“SMA doesn't define you as a person. You're more than just someone in a wheelchair.”
BACKGROUND MUSIC
Super: MORE STORIES OF HOPE
Maya, a teen with spinal muscular atrophy (SMA), shares her treatment journey
Sponsored by Biogen
MAYA: Hi, I’m Maya, I’m from Texas, and I’m a teen living with SMA.
Super: MAYA
An outgoing National Honor Society student and SMA advocate
Maya is a paid spokesperson for Biogen.
BACKGROUND MUSIC
Super: MORE STORIES OF HOPE
Maya, what inspires you to share your story?
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.
Super: “SMA doesn’t define you as a person”
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
Super: MORE STORIES OF HOPE
What has your experience been with SPINRAZA® (nusinersen)?
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: In clinical studies, the most common side effects of SPINRAZA included lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome (headache related to the intrathecal procedure).
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
Super: The CHERISH clinical trial studied 126 nonambulatory individuals aged 2 to 9 years with later-onset SMA.
At 15 months, the average score for people taking SPINRAZA showed a 3.9-point improvement in overall motor function, as measured by Hammersmith Functional Motor Scale-Expanded (HFMSE) versus a 1.0-point decline in the untreated control group.
BACKGROUND MUSIC
Super: “My motor skills have gotten better.”
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
Super: If you are considering treatment, talk to your doctor about SPINRAZA
SPINRAZA.com
[ISI present in video and available below.]
Super: ©2024 Biogen. All rights reserved. 04/24 SPZ-US-5747 225 Binney Street, Cambridge, MA 02142
BACKGROUND MUSIC
Super: MÁS HISTORIAS DE ESPERANZA
Maya, una adolescente con atrofia muscular espinal (AME), comparte su experiencia con el tratamiento
Patrocinado por Biogen
MAYA: Hola, soy Maya. Nací en Texas y soy una adolescente con AME.
Super: MAYA
Estudiante extrovertida de National Honor Society y defensora de la AME
Maya es portavoz remunerada de Biogen.
BACKGROUND MUSIC
Super: MÁS HISTORIAS DE ESPERANZA
Maya, ¿qué te motiva a compartir tu historia?
MAYA: Lo que me gustaría… que cambie en la mentalidad de la gente es… que la AME no te define como persona. Eres más que alguien en una silla de ruedas. Eso significa mucho para mí. Porque cada vez que la gente te ve, te dice: “Ah, sí, estás en silla de ruedas, te conozco”. Y no. Luego ven quién eres realmente cuando pasas tiempo con ellos y te dicen: “Eres realmente genial” Y es muy bonito
Super: La AME no te define comopersona. Eres más que solo una persona en un silla de ruedas.”
MAYA: Fui a la fiesta de bienvenida, en primer año, con mis amigos y ellos me ayudaron a colocarme el equipo en las rodillas y la parte superior. Me ubicaron para que pudiera levantarme y hacerme fotos con mis amigos y bailar con ellos... en mi silla, pero de pie, así que fue muy bonito.
MAYA: Mis amigos me cuidan y lo valoro mucho. Mi mamá no tiene que acompañarme a todas partes, salvo que sea algo muy difícil.
[ISI present in video and available below]
BACKGROUND MUSIC
Super: MÁS HISTORIAS DE ESPERANZA
¿Cómo ha sido tu experiencia con SPINRAZA® (nusinersen)?
MAYA: Empecé a usar SPINRAZA en marzo de 2017, cuando tenía nueve años. Lo estoy usando hace seis años. Ya tengo quince años, así que hace tiempo ya; y está muy bien.
Ten en cuenta que esta es mi experiencia con SPINRAZA. Para otras personas puede ser diferente.
Super: Los resultados individuales pueden variar en función de varios factores, como la gravedad de la enfermedad, el inicio del tratamiento y la duración de la terapia.
MAYA: En mis dosis iniciales, la primera vez, tuve un dolor de cabeza muy intenso, que me hizo vomitar la primera vez. Tuve dolores de cabeza durante todas esas dosis iniciales.
Super: En los estudios clínicos, los efectos secundarios más frecuentes de SPINRAZA incluyeron infección de las vías respiratorias bajas, fiebre, estreñimiento, dolor de cabeza, vómitos, dolor de espaida y syndrome posterior a la punción lumbar (dolor de cabeza relacionado con el procedimiento intratecal).
MAYA: SPINRAZA se aplica en una inyección en la parte baja de la espalda (inyección intratecal) y el medicamento se administra directamente en el sistema nervioso central. El tratamiento con SPINRAZA empieza con cuatro inyecciones en los primeros meses como dosis iniciales y luego se administra cada cuatro meses.
La primera vez que se usa SPINRAZA, se hacen análisis, según lo recomendado por un medico. Estos se repetirán antes de cada dosis para vigilar la sangre y los riñones. Consulte los riesgos y beneficios con su médico antes de empezar SPINRAZA.
BACKGROUND MUSIC
Super: El ensayo clínico CHERISH estudió a 126 personas no ambulatorias de 2 a 9 años con AME de aparición tardía.
A los 15 meses, la puntuación promedio para las personas que recibían SPINRAZA mostró una mejora de 3.9 puntos en la función motora general, medida por la Escala Motora Funcional Ampliada de Hammersmith (HFMSE), frente a una disminución de 1.0 puntos en el grupo de control no tratado.
BACKGROUND MUSIC
Super: “Mis habilidades motoras mejoraron”.
MAYA: Recuerdo que podía destapar los marcadores, algo que antes no podía hacer. Mis habilidades motoras mejoraron. Mi fuerza mejoró. Puedo levantar pesas de una libra, ya puedo hacerlo. No podía hacer eso antes, así que es muy importante para mí. Estoy agradecida de lo lejos que llegué.
BACKGROUND MUSIC
Super: Si considera este tratamiento, hable con su médico sobre SPINRAZA.
SPINRAZA.com
[ISI present in video and available below]
Super: ©2024 Biogen. Todos los derechos reservados. 05/24 SPZ-US-5846 225 Binney Street, Cambridge, MA 02142
LILY // AGE 7 // ASPIRING ARTIST
TREATED WITH SPINRAZA FOR 5+ YEARS
"There's help and there's hope."
Super:
BIOGEN LOGO
DANIEL (LILY’S DAD): Lily is intelligent, creative. One minute she's doing art for example. The next thing you know, she's going around playing with her brother. She keeps herself so occupied. She started playing the piano earlier this year. Taking weekly lessons. Growing up seemed pretty normal. But then her daycare teacher, age 2, noticed her motor functions. Something's a little off. How she picks things up off the floor. She can't bend her knees fully. But then when the teacher mentioned it again—ah okay, we'll go see a doctor. They said “We'll have to run some tests”. Ultimately she got diagnosed with SMA type 3.
LILY: Hi, I’m Lily. I have SMA.
Super:
DANIEL
LILY’S DAD
LILY
ON SPINRAZA
Sponsored by Biogen
Lily and her father are paid spokespersons for Biogen
DANIEL (LILY’S DAD): We were overwhelmed. Didn't know what we were going through.
We had hope because they had some options for us. The doctors did recommend SPINRAZA.
Super:
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
BACKGROUND MUSIC
FEMALE VOICE: WHAT IS SPINRAZA? SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.
Super:
WHAT IS SPINRAZA? SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.
SPINRAZA LOGO
SELECTED IMPORTANT SAFETY INFORMATION. A healthcare provider will monitor for signs of bleeding complications and kidney damage while taking SPINRAZA.
Super:
SELECTED IMPORTANT SAFETY INFORMATION
A healthcare provider will monitor for signs of bleeding complications and kidney damage while taking SPINRAZA.
SPINRAZA LOGO
These are not all the possible side effects. Please continue watching for additional Important Safety Information.
Super:
SELECTED IMPORTANT SAFETY INFORMATION
These are not all the possible side effects. Please continue watching for additional Important Safety Information.
SPINRAZA LOGO
DANIEL (LILY’S DAD): We decided to. You know, we didn’t wait. We’re like, let’s do it.
Super:
LILY’S SPIN on WHAT’S POSSIBLE
SPINRAZA LOGO
DANIEL (LILY’S DAD): What made us confident in our decision was that SPINRAZA has the longest clinical trial program in SMA.
Super: SPINRAZA (12 mg) clinical studies included patients from 3 days to 16 years of age at first dose and did not include sufficient numbers of subjects aged 65 and older to determine whether they respond differently from younger patients. Pivotal studies did not include adult patients.
SPINRAZA LOGO
DANIEL (LILY’S DAD): It's already being used by over 14,000 SMA patients worldwide. Lily's doing great. It's our story and it's unique to Lily’s journey.
Super: 14,000+ patients* have been treated with SPINRAZA worldwide, from 1 day to 85 years old.†
*Based on commercial patients and early access patients receiving treatment with SPINRAZA worldwide as of June 2024.
†Based on patients receiving treatment with SPINRAZA in the US as of May 2024.
SPINRAZA (12 mg) clinical studies included patients from 3 days to 16 years of age at first dose and did not include sufficient numbers of subjects aged 65 and older to determine whether they respond differently from younger patients. Pivotal studies did not include adult patients.
SPINRAZA LOGO
DANIEL (LILY’S DAD): Her motor skills are improving…
also on her 6-minute test. And I rely on the physical therapist because they actually have all the records and the test–the Hammersmith test.
Super: In a 15 month study in later-onset SMA, SPINRAZA group improved motor function compared to placebo group.
SPINRAZA LOGO
DANIEL (LILY’S DAD): She can go up the stairs now independently in her own Lily fashion, which before we always had to support her. And to see that is very encouraging, like she's come a long way.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
SPINRAZA LOGO
DANIEL (LILY’S DAD): 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 our 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.
Super: 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 our 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.
SPINRAZA LOGO
DANIEL (LILY’S DAD): I do depend a lot and rely upon the Family Access Manager that Biogen provides. She has our back. It takes the guesswork out of the logistics, the scheduling, the insurance, the co-pay assist. So I'd really like to encourage the families out there that they are not alone and there's help and there's hope.
[DANIEL LAUGHS]
Thanks…Lily!
[LILY LAUGHS]
DANIEL (LILY’S DAD): SPINRAZA was the route we decided to take and we’re glad we did so. Looking back to when she got diagnosed to today, wow, what a journey.
[ISI present in video and available below.]
Super: ©2025 Biogen. All rights reserved. SPZ-US-6042 03/25 225 Binney Street, Cambridge, MA 02142
Super: SPINRAZA LOGO
IF YOU’RE CONSIDERING TREATMENT, TALK TO YOUR DOCTOR ABOUT SPINRAZA
SPINRAZA.com
KIRRA & JUDE // AGE 5; MAVRIK // AGE 6
MORE STORIES OF HOPE
"It's very important to diagnose SMA early. The faster you know, the faster you react."
Super:
MORE STORIES OF HOPE
Parents of children living with SMA reflect on the significance of early diagnosis and power of early treatment
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.
Super:
In 2016, SPINRAZA® (nusinersen) was approved as a prescription medicine for infants, children and adults living with SMA
[Logo and ISI appear here]
These are stories of 3 families living with SMA. This is their experience. Others may have different experiences.
Super:
These are stories of 3 families living with SMA. This is their experience; others may have different experiences.
JUDE // AGE 5
KIRRA //AGE 5
MAVRIK // AGE 6
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.
Super: JENNIFER
Daughter Kirra (age 5) started treatment with SPINRAZA at 11 days old before SMA symptoms occurred
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: PEARL ANN
Son Mavrik (age 6) started treatment with SPINRAZA at 7 weeks old before SMA symptoms occurred
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: ALYSSA
Son Jude (age 5) started treatment with SPINRAZA at 9 days old before SMA symptoms occurred
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: In an ongoing supportive study, 25 infants who had not yet shown symptoms of SMA were treated with SPINRAZA for nearly 5 years.
The study had a small number of participants and no untreated group to compare results, so the data may not be as reliable as other clinical trial data.
Those were very tear-jerking milestones for us.
Super: IN THE STUDY: 100%
25 out of 25 were sitting without support after at least 14 months of taking SPINRAZA, and continued to do so after nearly 5 years.
Milestones we’re currently working on is walking without assistance.
Super: IN THE STUDY: 88%
22 out of 25 were walking with assistance after at least 14 months taking SPINRAZA.
Super: IN THE STUDY: 96%
24 out of 25 were walking with assistance after nearly 5 years.
JENNIFER (KIRRA’S MOTHER): Kirra’s doing a great job meeting her milestones. She’s sitting, walking. She is a joy to watch.
Super: IN THE STUDY: 77%
17 out of 22 were walking independently after at least 14 months taking SPINRAZA.
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.
Super: IN THE STUDY: 92%
23 out of 25 were walking independently after nearly 5 years.
Super: IN THE STUDY: 100%
25 out of 25 were alive without permanent ventilation after at least 14 months of taking SPINRAZA, and continued to do so after nearly 5 years.
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.
Super: SPINRAZA is given as an injection into the lower back, a procedure called intrathecal injection.
Treatment with SPINRAZA starts with four injections in the first two months as initial doses, and after that, SPINRAZA is given every four months.
Super: 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.
Super: SPINRAZA may increase the risk of bleeding and kidney damage. These are not all the possible side effects.
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.]
Super: ©2023 Biogen. All rights reserved. 11/23 SPZ-US-5527 225 Binney Street, Cambridge, MA 02142
FEMALE VOICEOVER: If you are considering treatment, talk to your child’s doctor about SPINRAZA.
Super: If you are considering treatment, talk to your child’s doctor about SPINRAZA.
SPINRAZA.com
CARLEE // AGE 11 // BAKER
TREATED WITH SPINRAZA FOR 6+ YEARS
"We are hopeful with our treatment with SPINRAZA."
Super: SPINRAZA logo
THIS MOMENT COMES FROM WITHIN
CARLEE // AGE 11
LATER-ONSET SMA
[Indication/ISI appear]
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super:
KELLI // MOTHER OF CARLEE
CARLEE HAS LATER-ONSET SMA
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.
Super:
CARLEE // AGE 11 // LATER-ONSET SMA
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.
Super: The initial 3 doses of SPINRAZA are administered at 14-day intervals, and the fourth dose is administered 30 days later.
Maintenance doses are administered every 4 months thereafter.
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.
Super: This monitoring is required before starting treatment and before each dose because SPINRAZA may increase the risk of bleeding and kidney damage.
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: THIS MOMENT COMES FROM WITHIN
This is Carlee’s moment.
What is yours?
[ISI present in video and available below.]
Super: ©2020 Biogen. All rights reserved. 06/20 SPZ-US-3231 225 Binney Street, Cambridge, MA 02142
ASHLEY // AGE 7 // BUDDING SCIENTIST
TREATED WITH SPINRAZA FOR 6+ YEARS
“The little gains mean so much.”
Super: SPINRAZA logo
THIS MOMENT COMES FROM WITHIN
ASHLEY // AGE 7
LATER-ONSET SMA
[Indication/ISI appear]
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super:
ANTONIA // MOTHER OF ASHLEY
ASHLEY HAS LATER-ONSET SMA
Ashley is an amazing little girl. She is curious about the world. She wants to know everything.
Super:
ASHLEY // AGE 7 // LATER-ONSET SMA
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: The initial 3 doses of SPINRAZA are administered at 14-day intervals, and the fourth dose is administered 30 days later.
Maintenance doses are administered every 4 months thereafter.
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.
Super: This monitoring is required before starting treatment and before each dose because SPINRAZA may increase the risk of bleeding and kidney damage.
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
Super: THIS MOMENT COMES FROM WITHIN
This is Ashley’s moment.
What is yours?
[ISI present in video and available below.]
Super: ©2020 Biogen. All rights reserved. 06/20 SPZ-US-3233 225 Binney Street, Cambridge, MA 02142
RUBY // AGE 4 IN VIDEO // BUTTERFLY PAINTER
“We feel like we have this tool to fight back with.”
Super: SPINRAZA logo
THIS MOMENT COMES FROM WITHIN
RUBY // AGE 4
LATER-ONSET SMA
[Indication/ISI appear]
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.
Super:
GINA // MOTHER OF RUBY
RUBY HAS LATER-ONSET SMA
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.
Super:
RUBY // AGE 4 // LATER-ONSET SMA
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.
Super:
STEVE // FATHER OF RUBY
RUBY HAS LATER-ONSET SMA
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.
Super: The initial 3 doses of SPINRAZA are administered at 14-day intervals, and the fourth dose is administered 30 days later.
Maintenance doses are administered every 4 months thereafter.
Super: This monitoring is required before starting treatment and before each dose because SPINRAZA may increase the risk of bleeding and kidney damage.
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: THIS MOMENT COMES FROM WITHIN
This is Ruby’s moment.
What is yours?
[ISI present in video and available below.]
Super: ©2021 Biogen. All rights reserved. 11/21 SPZ-US-1007 v3 225 Binney Street, Cambridge, MA 02142
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 // AGE 2 IN VIDEO // TU-TU LOVER
TREATED WITH SPINRAZA FOR 6+ YEARS
“She has got so much joy and so much love and so much drive.”
Super: SPINRAZA logo
THIS MOMENT COMES FROM WITHIN
SOFIA // AGE 2.5
EARLY-ONSET SMA
[Indication/ISI appear]
SOFIA’S SISTER: My sister Sofia is goofy, and she is also silly.
Super:
SOFIA // AGE 2.5 // EARLY-ONSET SMA
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.
Super:
JASON // FATHER OF SOFIA
SOFIA HAS EARLY-ONSET SMA
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.
Super:
NATALIA // MOTHER OF SOFIA HAS EARLY-ONSET SMA
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.
Super: The initial 3 doses of SPINRAZA are administered at 14-day intervals, and the fourth dose is administered 30 days later.
Maintenance doses are administered every 4 months thereafter.
Super: This monitoring is required before starting treatment and before each dose because SPINRAZA may increase the risk of bleeding and kidney damage.
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.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
Super: THIS MOMENT COMES FROM WITHIN
This is Sofia’s moment.
What is yours?
[ISI present in video and available below.]
Super: ©2021 Biogen. All rights reserved. 11/21 SPZ-US-0568 v5 225 Binney Street, Cambridge, MA 02142
"SMA doesn't define you as a person. You're more than just someone in a wheelchair."
"SMA doesn't define you as a person. You're more than just someone in a wheelchair."
“There’s help and there’s hope.”
"It's very important to diagnose SMA early. The faster you know, the faster you react."
"We are hopeful with our treatment with SPINRAZA."
“The little gains mean so much.”
“We feel like we have this tool to fight back with.”
“She has got so much joy and so much love and so much drive.”
*Based on commercial patients and early access patients receiving treatment with SPINRAZA worldwide as of June 2024.
†Based on patients receiving treatment with SPINRAZA in the US as of May 2024.
‡SPINRAZA (12 mg) clinical studies included patients from 3 days to 16 years of age at first dose and did not include sufficient numbers of subjects aged 65 and older to determine whether they respond differently from younger patients. Pivotal studies did not include adult patients.