All About Spinraza
If you have spinal muscular atrophy (SMA), your doctor may prescribe Spinraza. It’s a medication used to treat SMA in adults and children.
SMA is a group of rare genetic disorders. The motor neurons are in the spine. This leads to muscle weakness and muscle atrophy (wasting away of muscle).
To learn more about Spinraza’s use, see “Is Spinraza used for SMA?” below.
Spinraza basics
Nusinersen is the active ingredient in Spinraza. An active ingredient is what makes a drug work.
Spinraza comes as a vial of liquid solution. You’ll receive it as an intrathecal injection (an injection into the fluid surrounding the spinal cord). A specially trained healthcare professional will administer Spinraza to you at a designated treatment center.
“Read on to learn about Spinraza’s side effects.”
There are many factors that affect the cost of prescription drugs. What your insurance plan covers is one of the factors. Talk to your doctor about current prices for Spinraza injections. If you have questions about how to pay for your prescription, talk to them.
You may also want to enroll in a support program for Spinraza called SMA360. This program can help you learn about the financial assistance options available for the drug. To enroll, you’ll need to have your doctor fill out a start form. For more information, see the program webpage or call 844-477-4672.
You can also check out this article to learn more about saving money on prescriptions.
Find out what the answers are to some questions.
What is Spinraza’s mechanism of action?
Spinraza increases the levels of a certain proteins in the body to treat SMA.
People with spinal muscular atrophy (SMA) have low levels of a protein called SMN. This protein is important for proper functioning of nerve cells that control muscle movement.
Genes called SMN1 and SMN2 usually provide instructions that tell cells how to make SMN. In people with SMA, these instructions are missing or incomplete. Without enough SMN, nerve cells become damaged and die. This causes symptoms such as muscle weakness, muscle atrophy (wasting away of muscle), and trouble walking or breathing.
SMN levels in your body increase with Spinraza. It provides a missing piece in the instructions that tell your cells how to make SMN. Increased levels of SMN in your body may help with the symptoms of SMA.
Talk to your doctor about Spinraza.
How does Spinraza affect the life expectancy of someone with SMA type 1?
It’s not entirely clear. In studies, Spinraza treatment prolonged overall survival in children with SMA type 1. But the life expectancy of individuals receiving this treatment is unknown. (Life expectancy refers to how long someone is expected to live.)
SMA type 1 is the most common type. Symptoms usually start before 6 months. SMA type 1 children live less than 2 years without treatment.
There are studies to learn more about Spinraza and whether it might affect life expectancy. Your doctor can discuss the treatment with you.
How does Spinraza compare with Zolgensma and Evrysdi?
Spinraza, Zolgensma, and Evrysdi are all used to treat SMA. Spinraza and Evrysdi are used in adults and children of any age, while Zolgensma is used only in children younger than 2 years old.
The chart below shows some of the differences between drugs.
| Brand name | Active drug name | How it’s given | Usual dosing |
| Spinraza | nusinersen | intrathecal injection (an injection into the fluid surrounding the spinal cord) | once every 4 months (after receiving four starting doses) |
| Zolgensma | onasemnogene abeparvovec-xioi* | intravenous (IV) infusion (an injection into a vein given over time) | one-time dose |
| Evrysdi | risdiplam | a liquid solution that you swallow | once daily |
The reason for the drug\’s name being “-xioi” is to show that the drug is different from other drugs that may be created in the future.
Discuss the treatments with your doctor or pharmacist.
Is Spinraza a kind of gene therapy?
No, Spinraza isn’t a gene therapy. Gene therapy involves replacing a gene that isn’t working with one that does.
“Spinraza is a drug that has an antisense oligonucleotide in it. It works by increasing the levels of a single molecule of a single molecule of a single molecule of a single molecule of a single molecule of a single molecule of a single molecule of a single molecule of a single molecule of a single molecule of a single molecule of a single molecule of a single molecule Nerve cells control muscle movement. People with SMA don’t have enough SMN.”
Your doctor can tell you more about the options for your condition.
Does Spinraza cause hydrocephalus?
Hydrocephalus wasn’t seen in Spinraza’s studies. Hydrocephalus occurs when fluid collects in the brain, causing the head to increase in size.
Some people who have received Spinraza have reported this condition. The drug became available for use. It is not known how often it happened or if it was caused by other factors.
Talk to your doctor about the risk of hydrocephalus with Spinraza treatment.
Your doctor will explain how Spinraza works. They will explain how much you will get and how often.
Before starting Spinraza treatment, your doctor will have you get blood tests to check the health of your kidneys and the level of platelets (a type of blood cell). You’ll repeat these blood tests before each injection. The results of these tests will help your doctor determine if it’s safe for you to receive the drug.
A specially trained healthcare professional will administer Spinraza to you at a designated treatment center. You cannot give yourself this drug at home.
Spinraza is a liquid solution. Its route of administration is not straight. This means a healthcare professional injects the drug into your spine.
How a Spinraza injection is given
A healthcare professional will administer Spinraza as an intrathecal injection. This procedure is also called a lumbar puncture.
The basic steps of Spinraza administration are listed here.
- A healthcare professional will help you get into the right position. You can be sitting on your side or lying on your front.
- They’ll choose an area with no signs of infection or inflammation and use a disinfecting solution to clean the skin on your lower back. They may also give you medications, such as sedatives and local anesthetics. (Sedatives make you feel calm and sleepy. Local anesthetics help to numb your skin at the injection site.)
- The healthcare professional will insert a needle into your lower back. They may use ultrasound or other imaging techniques to help guide the needle into the space between two bones in your lower spine.
- They will remove a small amount ofCSF once they place the needle. CSF is the fluid that surrounds your brain.
- The healthcare professional will inject Spinraza.
The manufacturer provides more details about this procedure. If you have questions about how Spinraza is given, talk with your doctor.
Questions for your doctor
You may have questions about Spinraza. Discuss your concerns with your doctor.
Here are a few tips that might help you.
- Before your appointment, write down questions such as:
- How will Spinraza affect me?
- Bring someone with you to your appointment to make you feel more comfortable.
- “If you don’t understand something, ask your doctor to explain it.”
“Your doctor and other healthcare professionals are available to help you. They want you to get the best care possible. Don’t be afraid to ask questions or give feedback on your treatment”
Spinraza is used to treat spinal muscular atrophy (SMA) in adults and children of any age.
About SMA and its types
SMA is a group of rare genetic disorders. The motor neurons are in the spine.
People with SMA have low levels of aprotein. Motor neurons work with thisProtein.
Genes called SMN1 and SMN2 usually provide instructions that tell your cells how to make SMN. In people with SMA, these instructions are missing or incomplete. Without enough SMN, motor neurons start to die. This causes symptoms such as muscle weakness, muscle atrophy (wasting away of muscle), and trouble walking or breathing. These symptoms usually worsen over time.
There are different types of SMA. SMA types vary in their usual start.
- SMA type 1: Symptoms start before the age of 6 months.
- SMA type 2: Symptoms appear between the ages of 6 to 18 months.
- SMA type 3: Symptoms start after the age of 18 months.
- SMA type 4: Symptoms can begin at age 18 years but usually appear after age 35 years.
SMA types 1 and 2 are sometimes called infant-onset SMA, while SMA types 3 and 4 are sometimes called later-onset SMA. SMA is also called type 4.
About Spinraza for SMA
SMN levels in your body increase with Spinraza. It provides a missing piece in the instructions that tell your cells how to make SMN. Increased levels of SMN in your body may help with the symptoms of SMA.
Talk to your doctor about Spinraza and its effectiveness for SMA.
Your doctor will tell you the right amount of Spinraza. Your doctor will let you know your dose schedule.
Before starting Spinraza treatment, your doctor will have you get blood tests to check the health of your kidneys and the level of platelets (a type of blood cell). You’ll repeat these blood tests before each injection. The results of these tests will help your doctor determine if it’s safe for you to receive the drug.
Form
Spinraza comes as a vial of liquid solution. You’ll receive it as an intrathecal injection (an injection into the fluid surrounding the spinal cord). A specially trained healthcare professional will administer Spinraza to you at a designated treatment center.
Recommended dosages
The treatment begins with four loading doses. A loading dose is a higher dose of a medication. If you received a lower initial dose, the drug would not work as well.
You will receive the second dose 14 days after the first one. You will wait another 14 days for the third. The third and fourth loading dose are given within 30 days.
You will likely receive the maintenance dose of Spinraza after the loading doses. This is usually a single dose.
Questions about Spinraza’s dosage
There are many questions about Spinraza.
- What if I miss a dose of Spinraza? If you miss an appointment to receive Spinraza, contact the treatment center right away. Someone there will help you reschedule your injection as soon as possible.
- Will I need to use Spinraza long term? If you and your doctor decide that Spinraza is working well for you, you’ll likely use it long term.
- How long does Spinraza take to work? Spinraza starts working as soon as you receive your first dose. But it may take a few months before you notice any improvement in your muscle movement.
If you have questions about Spinraza, ask your doctor.
Spinraza may cause mild or serious side effects. Some of the more common side effects of Spinraza are described below. All possible side effects are not included in these lists.
Side effects of a drug can depend on other factors.
- Your age.
- You have other health conditions.
- You take other drugs.
“The doctor or pharmacist can tell you more about Spinraza’s side effects. They can suggest ways to reduce side effects.”
Mild side effects
Here’s a short list of some of the mild side effects that Spinraza can cause. To learn about other mild side effects, talk with your doctor or pharmacist, or read Spinraza’s prescribing information.
These side effects may vary depending on when the spinal muscular atrophy (SMA) began. (SMA is the condition that Spinraza is used to treat.)
There are some mild side effects of Spinraza that have been reported.
- lower respiratory infection, such as bronchitis
- It is a problem of the colon.
- There is a high degree of fever.
- There is a throbbing head.
- vomiting
- Back pain.
- There is a rash.
- mild allergic reaction*
The side effect is described in the section “Allergic reaction”.
The side effects of many drugs can be gone in a few days. If they become intolerable, talk to your doctor or pharmacist.
Serious side effects
Spinraza can have serious side effects. If you think you have developed serious side effects from Spinraza, call your doctor. If you think you have a medical emergency, you should call the emergency number.
Spinraza has been reported to have serious side effects.
- There are problems with the kidneys.
- low level of platelets (a type of blood cell) and abnormal blood test results, both of which may indicate an increased risk of bleeding
- There is a severe allergic reaction.
The side effect is described in the section “Allergic reaction”.
Allergic reaction
Some people may have an allergic reaction to Spinraza. Allergic reaction wasn’t seen in Spinraza’s studies but has been reported since the drug became available for use.
A mild allergic reaction can include some symptoms.
- skin There is a rash.
- It is itchy.
- flushing (temporary warmth, redness, or deepening of skin color)
A more severe allergic reaction is not uncommon. A severe allergic reaction can cause swelling under your skin, usually in your lips, hands, or feet. They can include swelling of your mouth, throat, and tongue, which can cause trouble breathing.
If you have an allergic reaction to Spinraza, call your doctor. If you think you have a medical emergency, call the emergency number.
This section covers important things to discuss with your doctor when you are considering treatment with Spinraza.
Interactions
Taking a medication with certain vaccines, foods, and other things can affect how the medication works. These effects are called interactions.
Before taking Spinraza, you should tell your doctor about all your medication. You should describe any vitamins, herbs, or supplements you use. Your doctor or pharmacist can tell you about any interactions that may occur.
The “Warnings” section contains information about drug-condition interactions.
Interactions with drugs or supplements
There are no known drugs or supplements that interact with Spinraza. Drug interactions with Spinraza will be recognized in the future. New medications may be approved that interact with Spinraza.
You should still tell your doctor and pharmacist about any other drugs you take. They can check for new interactions during your treatment.
Warnings
If you have certain medical conditions, Spinraza may not be right for you. These are drug-condition interactions. Spinraza is a good treatment option if you have other factors to consider.
Before you take Spinraza, you should talk to your doctor about your health history. The factors to consider are listed below.
- Allergic reaction. If you’ve had an allergic reaction to Spinraza or any of its ingredients, your doctor will likely not prescribe Spinraza. Ask them what other medications are better options for you.
Spinraza and alcohol
Spinraza and alcohol have no known interactions. If you drink alcohol, you should talk to your doctor about how much you can drink during treatment.
Pregnancy and breastfeeding
Spinraza is not known if it is safe for use during a pregnant or breastfeeding period.
If you are pregnant or planning a baby, you should talk to your doctor about the risks and benefits of taking this medication.
Talk to your doctor if you are planning to breastfeeding. They can help you decide how to feed your child.
Spinraza can be an effective treatment for spinal muscular atrophy (SMA). If you and your doctor are considering Spinraza as a treatment option, you may have questions. It’s important to talk with your doctor about any questions or concerns you have before starting Spinraza treatment. Some examples include:
- How will I know if this medication is working?
- Can I take over-the-counter pain killers?
- Will I need to receive Spinraza treatment?
There are articles about SMA treatment options and resources for support.
- Treatments for the disease of the spine.
- “Resource that can help with your child’s diagnosis of SMA.”
Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.
