RESOURCE LIBRARY / VIDEO HUB
Hear from experts, patients living with SMA, and their families
Expert Views
Hear from experts in SMA.
Reinitiating SPINRAZA
Drs Nassim Rad and Crystal Proud discuss reinitiating patients on SPINRAZA and their clinical experience.
Transcript
Reinitiating SPINRAZA® (nusinersen)
Speaker
Text on screen
Indication and Important Safety Information. INDICATION
SPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
Continue watching for additional Important Safety Information.
INDICATION
SPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION
Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
Continue watching for additional Important Safety Information.
Hundreds of patients with SMA have returned to SPINRAZA
This is sponsored by Biogen®. Speakers are being compensated by Biogen for their time spent delivering this presentation.
SMA, spinal muscular atrophy.
Nassim Rad, MD, Director, SMA Multidisciplinary Care, University of Washington Medical Center:
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.
Nassim Rad, MD Director, SMA Multidisciplinary Care University of Washington Medical Center
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
SMA, spinal muscular atrophy.
Talk with your patients about whether their SMA therapy is achieving their treatment goals
SMA, spinal muscular atrophy.
Crystal Proud, MD, Pediatric Neuromuscular Neurologist, Children’s Specialty Group and Children’s Hospital of The King’s Daughters:
The communication strategy often involves addressing patient concerns, emphasizing the importance of treatment, and fostering an informed decision-making process to ensure that patients feel informed and involved in their care.
Crystal Proud, MD Pediatric Neuromuscular Neurologist Children's Specialty Group and Children's Hospital of the King's Daughters
In terms of safety, inform patients and caregivers that SPINRAZA can increase the risk of bleeding and could cause renal toxicity. Inform them of the importance of obtaining blood laboratory testing and urine testing at baseline and prior to each dose.
SELECTED IMPORTANT SAFETY INFORMATION
Inform patients and caregivers that SPINRAZA can increase the risk of bleeding and could cause renal toxicity. Inform them of the importance of obtaining blood laboratory testing and urine testing at baseline and prior to each dose.
Please see additional Important Safety Information later on in this video.
Reinitiating SPINRAZA after missed doses
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.
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.
Welcome your patients back to SPINRAZA
Crystal Proud, MD:
I’ve had patients reinitiate SPINRAZA. I would encourage my peers to reach out to their Biogen representative, who can share resources to help support patients with this transition.
To learn more about reinitiating SPINRAZA, visit spinrazahcp.com.
Important Safety Information presented in video and available below.
©2026 Biogen. All rights reserved.
225 Binney Street, Cambridge, MA 02142
SPZ-US-6008v3 06/26
Redefine What’s Possible
Drs Perry Shieh and Crystal Proud highlight the evidence for SPINRAZA in clinical practice, including the impact they have experienced in their practices.
Transcript
Redefine What’s Possible
Speaker
Text on screen
Indication and Important Safety Information. INDICATION
SPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
Continue watching for additional Important Safety Information.
INDICATION
SPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION
Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
Continue watching for additional Important Safety Information.
Perry Shieh, MD, PhD, Clinical Professor, Neurology and Pediatrics, University of California, Los Angeles:
With the breadth of clinical data spanning different patient types, SPINRAZA offers established efficacy and safety for individuals living with SMA.
Perry Shieh, MD, PhD Clinical Professor, Neurology and Pediatrics University of California, Los Angeles
This is sponsored by Biogen®. Speakers are being compensated by Biogen for their time to deliver this presentation.
SMA, spinal muscular atrophy.
Crystal Proud, MD, Pediatric Neuromuscular Neurologist, Children’s Specialty Group and Children’s Hospital of The King’s Daughters:
I remember exactly where I was when SPINRAZA was approved. It was around the holidays. And my phone just instantly blew up with messages and phone calls.
Perry Shieh, MD:
I went into this field to help people and for many years I didn’t always feel like I was helping patients. But after I started treating them with SPINRAZA, some of them started improving. I felt like I was helping people and that means a lot.
Crystal Proud, MD Pediatric Neuromuscular Neurologist, Children's Specialty Group and Children's Hospital of The King's Daughters
This is sponsored by Biogen. Speakers are being compensated by Biogen for their time to deliver this presentation.
CHERISH: The first clinical trial in later-onset SMA with proven results.
Crystal Proud, MD:
My goal is to empower patients with a clear understanding of SPINRAZA’s efficacy and safety data. This helps them understand that SPINRAZA has the potential to preserve or in some cases improve motor function.
Perry Shieh, MD:
The efficacy and safety of SPINRAZA have been studied in later-onset SMA in the CHERISH study, which was a phase 3, randomized, double-blind, sham-controlled, multicenter trial.
SMA, spinal muscular atrophy.
Crystal Proud, MD:
126 patients with SMA type 2 or type 3 were included in this study. The median age at screening was 3 years and the range was 2 to 9 years.
SPINRAZA Low Dose Regimen: 84 patients
Sham-control: 42 patients
SMA, spinal muscular atrophy.
Crystal Proud, MD:
The primary endpoint was least-squares mean change from the baseline in the HFMSE total score at month 15. Mean score for patients treated with SPINRAZA was a 3.9-point increase. And sham-control group was one point mean decrease in Hammersmith scores.
Primary outcome:
Least-squares mean change from baseline in HFMSE total score at 15 months.
Continue listening for additional Important Safety Information, and see the full Prescribing Information via the link provided.
HFMSE, Hammersmith Functional Motor Scale—Expanded.
Crystal Proud, MD:
In terms of safety, inform patients and caregivers that SPINRAZA can increase the risk of bleeding and could cause renal toxicity. Inform them of the importance of obtaining blood laboratory testing and urine testing at baseline and prior to each dose.
The most common adverse reactions that occurred in at least 20% of SPINRAZA-treated patients and occurred at least 5% more frequently than in sham-control patients were fever, headache, vomiting, and back pain.
SELECTED IMPORTANT SAFETY INFORMATION
Inform patients and caregivers that SPINRAZA can increase the risk of bleeding and could cause renal toxicity. Inform them of the importance of obtaining blood laboratory testing and urine testing at baseline and prior to each dose.
Please see additional Important Safety Information later on in this video.
Perry Shieh, MD:
I’ve seen my patients' journey with SPINRAZA. The breadth of SPINRAZA clinical data supports what I’ve experienced firsthand.
I have patients who stay on SPINRAZA because they’ve had a good response in terms of motor function. They see themselves able to do things that they weren’t able to do before. So they’re grateful for a therapy that has worked for them.
Continue listening for additional Important Safety Information, and see the full Prescribing Information via the link provided.
Making a difference for patients
Crystal Proud, MD:
It is incredibly impactful to be able to provide a treatment that is meaningful to my patients’ lives and impacts their ability to do things that we might consider simple, but are incredibly valuable to them.
Perry Shieh, MD:
Well, SPINRAZA has really changed the practice of taking care of SMA patients. It’s given them hope and it’s given us hope. And that’s probably the most important ingredient.
SMA, spinal muscular atrophy.
Crystal Proud, MD:
So it’s been incredible to be able to see them really shine in an era of treatment.
Patients treated with SPINRAZA in the US have a collective ~16,700 years of treatment experience.* Learn more at SPINRAZAhcp.com
*Based on consented patients from a real-world sample in the US who are or have ever been treated with SPINRAZA through April 2026.
Important Safety Information presented in video and available below.
©2026 Biogen. All rights reserved.
225 Binney Street, Cambridge, MA 02142
SPZ-US-6027v3 06/26
An ASO Approach to SMA
Drs Crystal Proud and Chamindra Laverty talk about SPINRAZA, an antisense oligonucleotide (ASO) treatment, and their approach to talking with patients.
Transcript
An Antisense Oligonucleotide (ASO) Approach
Speaker
Text on screen
Indication and Important Safety Information. INDICATION
SPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
Continue watching for additional Important Safety Information.
INDICATION
SPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION
Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
Continue watching for full Important Safety Information.
TARGETING AN UNDERLYING CAUSE OF SMA
SPINRAZA is designed to target a specific sequence in SMN2 pre-mRNA
mRNA, a messenger ribonucleic acid; SMA, spinal muscular atrophy; SMN2, survival motor neuron 2 gene.
TARGETING AN UNDERLYING CAUSE OF SMA to specifically modify splicing, which increases exon 7 inclusion and production of full-length SMN protein
SMA, spinal muscular atrophy; SMN, survival motor neuron.
The first and only ASO treatment for SMA
ASO, antisense oligonucleotide; SMA, spinal muscular atrophy
Crystal Proud, MD, Pediatric Neuromuscular Neurologist, Children’s Specialty Group and Children’s Hospital of The King’s Daughters:
So the motor neurons that are involved in spinal muscular atrophy sit in the anterior portion of the spinal cord.
Crystal Proud, MD Pediatric Neuromuscular Neurologist Children's Specialty Group and Children's Hospital of The King's Daughters
This is sponsored by Biogen. Speakers are being compensated by Biogen for their time spent delivering this presentation.
SPINRAZA is directly delivered to the CNS via an intrathecal injection, which bypasses the blood-brain barrier.
Directly delivered to the CNS via an intrathecal injection
CNS, central nervous system.
And this allows SPINRAZA to be distributed from the CSF to target the CNS tissues where it increases the production of full-length SMN protein.
Targets CNS tissues
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
CNS, central nervous system; CSF, cerebrospinal fluid; SMN, survival motor neuron.
So by administering SPINRAZA into the intrathecal space it can impact those tissues that it needs to impact.
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
Maintains high concentration and long half-life in the CSF (~135-177 days)
CSF, cerebrospinal fluid.
Intrathecal delivery of SPINRAZA may be possible for many people with SMA
SMA, spinal muscular atrophy
Infants. Children. Teens. Adults.
Given by, or under the direction of, a healthcare professional experienced in performing lumbar punctures.
Chamindra Laverty, MD, Neuromuscular Specialist, Rady Children’s Hospital-San Diego:
I think probably the most memorable case is a small child. She was a little over two years old and I had to do lumbar punctures on her when she was still awake. And she just rose to the occasion every single time. It was lovely. And the entire staff loved having her come back for her SPINRAZA injections.
Chamindra Laverty, MD Neuromuscular Specialist Rady Children's Hospital-San Diego
Crystal Proud, MD:
In terms of safety, inform patients and caregivers that SPINRAZA can increase the risk of bleeding and could cause renal toxicity. Inform them of the importance of obtaining blood laboratory testing and urine testing at baseline and prior to each dose.
SELECTED IMPORTANT SAFETY INFORMATION
Inform patients and caregivers that SPINRAZA can increase the risk of bleeding and could cause renal toxicity. Inform them of the importance of obtaining blood laboratory testing and urine testing at baseline and prior to each dose.
Please see additional Important Safety Information later on in this video.
Guiding treatment conversations with adult patients
Crystal Proud, MD:
I think many of us have experienced this where a patient comes in and already has identified what they think that they need. I think it's important that we then go back to the basics once again; discuss the biology of their diagnosis, set the foundation, discuss options for intervention, and at that point we can incorporate what they think that they know about the particular therapy.
And then I tend to take it a step further and say, "My recommendation for you would be this particular treatment and here's why. Here's how we would go about facilitating your care with this therapy.”
A DIVERSE SMA COMMUNITY
Patients treated with SPINRAZA in the US have a collective ~17,600 years treatment experience*
*Based on consented patients from a real-world sample in the US who are or who have ever been treated with SPINRAZA through April 2026.
SMA, spinal muscular atrophy.
A DIVERSE SMA COMMUNITY
Ranging from 0 days to 86 years old*†
*Based on patients receiving treatment with SPINRAZA in the US as of April 2026.
†Pivotal studies did not include adult patients. Clinical studies did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently than younger subjects.
SMA, spinal muscular atrophy.
Crystal Proud, MD:
SPINRAZA has dramatically changed the way that I practice and care for patients with spinal muscular atrophy.
The first-ever FDA-approved treatment for people with SMA.
Learn more at SPINRAZAhcp.com.
Important Safety Information presented in video and available below.
©2026 Biogen. All rights reserved.
225 Binney Street, Cambridge, MA 02142
SPZ-US-6028v3 06/26
Considerations for Treatment-Naive Adults
Drs Crystal Proud, Chamindra Laverty, and Perry Shieh share their approach to treatment-naive adults, how they talk with a patient who may be hesitant, and the importance of starting treatment.
Transcript
Considerations for Treatment-Naive Adults
Speaker
Text on screen
Indication and Important Safety Information. INDICATION
SPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
Continue watching for additional Important Safety Information.
INDICATION
SPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION
Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
Continue watching for full Important Safety Information.
BASED ON 2021 US CLAIMS DATA N=353
~78% of surveyed adults with SMA have never received treatment
This is sponsored by Biogen. Speakers are being compensated by Biogen for their time spent delivering this presentation.
Claims from 01 January 2016 through 30 September 2022 for US patients with SMA.
SMA, spinal muscular atrophy.
How do you offer them options?
This is sponsored by Biogen. Speakers are being compensated by Biogen for their time spent delivering this presentation.
Crystal Proud, MD, Pediatric Neuromuscular Neurologist, Children’s Specialty Group and Children’s Hospital of The King’s Daughters:
It’s likely that treatment-naive adult patients with SMA have been living with their disease for a long time and may have developed coping mechanisms. So they may resist treatment due to a combination of factors, including fear of potential side effects, concerns about the impact on their daily routines, and a lack of understanding about the benefits of treatment.
Because of these factors, it is important to educate treatment-naive adults on the natural progression of SMA and showcase the treatment options available to them.
Crystal Proud, MD Pediatric Neuromuscular Neurologist Children's Specialty Group and Children's Hospital of The King's Daughters
SMA, spinal muscular atrophy.
Chamindra Laverty, MD, Neuromuscular Specialist, Rady Children’s Hospital-San Diego:
Sometimes loss of motor function can be so gradual that adults living with SMA may not understand the progressive nature of this disease.
The most important thing when trying to speak to an adult patient with mild SMA is to try to help them project to the future to see, to help them realize what it might look like in the future.
Chamindra Laverty, MD Neuromuscular Specialist Rady Children's Hospital-San Diego
SMA, spinal muscular atrophy.
SPINRAZA® experience
Crystal Proud, MD:
In terms of safety, inform patients and caregivers that SPINRAZA can increase the risk of bleeding and could cause renal toxicity. Inform them of the importance of obtaining blood laboratory testing and urine testing at baseline and prior to each dose.
SELECTED IMPORTANT SAFETY INFORMATION
Inform patients and caregivers that SPINRAZA can increase the risk of bleeding and could cause renal toxicity. Inform them of the importance of obtaining blood laboratory testing and urine testing at baseline and prior to each dose.
Please see additional Important Safety Information later on in this video.
Crystal Proud, MD:
The efficacy and safety of SPINRAZA have been studied in later-onset SMA in the CHERISH trial, which was a phase 3, randomized, double-blind, sham-controlled, multicenter trial.
SMA, spinal muscular atrophy.
Perry Shieh, MD, PhD, Clinical Professor, Neurology and Pediatrics, University of California, Los Angeles: To date, SPINRAZA has the largest number of real-world evidence studies in adults with SMA.
As of January 2024 SPINRAZA (12 mg) has the most real-world evidence in adults with SMA
SMA, spinal muscular atrophy.
Real-world evidence studies help supplement our understanding of the efficacy and safety of a drug in a wider population outside of a clinical trial.
Perry Shieh, MD, PhD Clinical Professor, Neurology and Pediatrics University of California, Los Angeles
Chamindra Laverty, MD:
SPINRAZA for me in my practice equates to having an option, an option for my patients to help them with their weakness—and it is wonderful to have choices in a world that had no choices prior to SPINRAZA.
5,300+ adults have been treated with SPINRAZA worldwide*†
*Based on commercial patients and early access patients receiving treatment with SPINRAZA as of June 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, so could not determine whether they would respond differently from younger patients. Pivotal studies did not include adult patients.
Learn more about the extensive data and results for SPINRAZA at SPINRAZAhcp.com.
Important Safety Information presented in video and available below.
©2026 Biogen. All rights reserved.
225 Binney Street, Cambridge, MA 02142
SPZ-US-6029v3 06/26
Reinitiating SPINRAZA
Reinitiating SPINRAZA
Drs Nassim Rad and Crystal Proud discuss reinitiating patients on SPINRAZA and their clinical experience.
Redefine What’s Possible
Redefine What’s Possible
Drs Perry Shieh and Crystal Proud highlight the evidence for SPINRAZA in clinical practice, including the impact they have experienced in their practices.
An ASO Approach to SMA
An ASO Approach to SMA
Drs Crystal Proud and Chamindra Laverty talk about SPINRAZA, an antisense oligonucleotide (ASO) treatment, and their approach to talking with patients.
Considerations for Treatment-Naive Adults
Considerations for Treatment-Naive Adults
Drs Crystal Proud, Chamindra Laverty, and Perry Shieh share their approach to treatment-naive adults, how they talk with a patient who may be hesitant, and the importance of starting treatment.
SMA, spinal muscular atrophy.
Hear from Patients
Meet patients and families living with spinal muscular atrophy (SMA) and listen to their SPINRAZA stories.
3 Families, 3 Stories // Presymptomatic SMA
SPINRAZA stories in patients with SMA who were treated with SPINRAZA before symptoms presented.
Transcript
3 Families, 3 Stories // Presymptomatic SMA
Speaker
Text on screen
INDICATION
SPINRAZA® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION
Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.
Continue watching for full Important Safety Information
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.
These are stories of three families living with SMA. This is their experience...
...Others may have different experiences.
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.
Jennifer
Daughter Kirra (age 5) started treatment with Low Dose 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.
Patient was on Low Dose SPINRAZA when photographed.
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.
Pearl Ann
Son Mavrik (age 6) started treatment with Low Dose 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.
Patient was on Low Dose SPINRAZA when photographed.
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.
Alyssa
Son Jude (age 5) started treatment with Low Dose 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.
Patient was on Low Dose SPINRAZA when photographed.
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.
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 had recommended SPINRAZA, and he received his first dose of SPINRAZA at 7-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.
In an 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.
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.
IN THE STUDY: 88%
22 out of 25 were walking with assistance after at least 14 months taking SPINRAZA.
IN THE STUDY: 96%
24 out of 25 were walking with assistance after nearly 5 years.
JENNIFER (KIRRA’S MOTHER): Kirra is doing a great job meeting her milestones. She’s sitting, walking. She is a joy to watch.
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.
IN THE STUDY: 92%
23 out of 25 were walking independently after nearly 5 years.
JENNIFER (KIRRA’S MOTHER): We didn't know. 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.
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.
SPINRAZA is given as an injection into the lower back. A procedure called an intrathecal injection, delivering medication directly into the central nervous system.
SPINRAZA Low Dose Regimen
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...
SPINRAZA Low Dose Regimen
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.
SPINRAZA may increase the risk of bleeding and kidney damage. These are not all the possible side effects.
Patients were on Low Dose SPINRAZA when photographed.
PEARL ANN (MAVRIK’S MOTHER): I want other parents to know they’re not alone.
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
Patients were on Low Dose SPINRAZA when photographed.
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.
Important Safety Information presented in video and available below.
©2026 Biogen. All rights reserved.
225 Binney Street, Cambridge, MA 02142
SPZ-US-5912v2 06/26
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
3 Families, 3 Stories // Presymptomatic SMA
3 Families, 3 Stories // Presymptomatic SMA
SPINRAZA stories in patients with SMA who were treated with SPINRAZA before symptoms presented.
Hear some of these patients’ stories
From children to adults, every SMA patient has a unique journey. But these patients have one thing in common—they chose SPINRAZA.
SMA, spinal muscular atrophy.