An Important Day in Treating SMA
2016 was shaping up to be a wonderful holiday season for Christine and her two sons, Caleb and Camden, who have spinal muscular atrophy (SMA). Despite having no treatment options available, at the time, she was determined and fearless as ever in her pursuit to give her sons the best life they could have. What happened next gave Christine and the SMA community hope.
Five years ago, SPINRAZA® (nusinersen) made history as the first FDA-approved prescription medicine used to treat SMA in pediatric and adult patients. When Christine’s older son, Caleb, was diagnosed with SMA years earlier, there wasn’t a treatment available but shortly after Camden’s birth and diagnosis, she was happy to learn there was a treatment option and he was treated presymptomatically.
“We are very thankful for the advances in science and what’s available to Camden and our family,” she said.
Dr. Perry Shieh is a practicing neurologist and professor, who was actively involved in conducting the ENDEAR clinical trial that supported the approval of SPINRAZA. He recalls his impressions of the SPINRAZA approval.
“As a doctor, it’s hard when all we can do is offer suggestions to patients to help live with the disease but not really treat it. SPINRAZA was a much-needed sign of hope,” Dr. Shieh said. “Of course, individual results may vary, e.g., with disease severity, initiation/duration of treatment.”
Because SMA affects children through adults with varying levels of severity, SPINRAZA’s approval was even more meaningful to the SMA community because it was approved for a broad range of patients.
SPINRAZA is administered intrathecally by, or under the direction of, healthcare professionals experienced in performing lumbar punctures. SPINRAZA (recommended dosage of 12 mg [5mL] per administration) begins with four initial loading doses; after the loading doses, SPINRAZA is administered every four months.
For all patients on SPINRAZA, ongoing monitoring is necessary to check for bleeding and kidney damage. Some of the common side effects of SPINRAZA include lower respiratory infection, fever, headache, vomiting, back pain, and post-lumbar puncture syndrome.
For adult patients like Robb, SPINRAZA’s approval restored a sense of possibility among those who thought there may never be a treatment option available to them.
Robb, who was diagnosed with SMA Type 2 as a child, barely had enough upper body strength to hold a knife and fork and even struggled to drive his wheelchair. When he learned about SPINRAZA from his sister, he started on treatment after talking to his neurologist about its potential benefits and risks.
“Before SPINRAZA, I couldn’t hold a pencil,” he said. “Now, I can hold a pencil. When I go in for treatment, the nurse tests my ability to hold a pencil and draw a line through a maze. While technically, I still fail the test, I try to improve every time and smile because this is my moment of strength, my moment to control the pencil to the best of my ability.”
Robb was excited to share, “I’m thrilled that, today, treatment options are available for people with SMA. Most kids born with SMA have options and hope like many of us never had.”
One of those kids is Sofia, who was diagnosed with early-onset SMA as a baby. Her mother Natalia recalled that “after treatment and lots of physical therapy, practice, and repetition, Sofia started lifting both her legs.”
Sofia still needs assistance and uses a motorized wheelchair, but she remains as encouraged as ever despite her SMA diagnosis. She is even enrolled in ballet classes and loves buzzing around in her walker with the other dancers.
Behind every SMA diagnosis, there is a unique journey filled with strength, resilience, and perseverance. Since its approval, more than 11,000 people have been treated with SPINRAZA worldwide, and each of those victories is personal.1
To learn more about SPINRAZA and what it has meant to people living with SMA and their loved ones, click here.
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.
These are not all the possible side effects of SPINRAZA. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Before taking SPINRAZA, tell your healthcare provider if you are pregnant or plan to become pregnant.
Please see full Prescribing Information.
This information is not intended to replace discussions with your healthcare provider.
Please see our website SPINRAZA.com for more information.
- Based on commercial patients, early access patients, and clinical trial participants through December 2020.