Spinraza is a medication used to treat spinal muscular atrophy (SMA) in children and adults. Spinraza is given by injection into the spinal fluid.

Spinraza Overview

Reviewed: March 7, 2017

Spinraza is a prescription medication used to treat spinal muscular atrophy (SMA) in children and adults. 

SMA is caused by genetic mutations that lead to a deficiency in a protein called survival motor neuron (SMN), an essential protein to motor neurons. Spinraza is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide. It is designed to target the mutated gene and increase production of the full-length SMN protein, which is critical for the maintenance of motor neurons.

Spinraza is available as a solution for injection that must be administered by a trained healthcare professional. It is given by intrathecal injection into the fluid surrounding the spinal cord.

Common side effects include upper and lower respiratory infection, constipation, headache, backpain and post-lumbar puncture syndrome.

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Spinraza Cautionary Labels


Uses of Spinraza

Spinraza is a prescription medication used to treat spinal muscular atrophy (SMA) in pediatric and adult patients. 

This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.



For more information on this medication choose from the list of selections below.

Side Effects of Spinraza

Serious side effects have been reported with Spinraza. See the “Spinraza Precautions” section. 

Common side effects of Spinraza include the following:

  • Lower respiratory infection
  • Upper respiratory infection
  • Constipation
  • Headache
  • Back pain
  • Post-lumbar puncture syndrome

This is not a complete list of Spinraza side effects. Ask your doctor or pharmacist for more information. Tell your doctor if you have any side effect that bothers you or that does not go away. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.


Spinraza Interactions

No drug interactions have been determined by the manufacturer. However, you should tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements. Not all drug interactions are known or reported and new drug interactions are continually being reported. 

Spinraza Precautions

Serious side effects have been reported with Spinraza including the following:

Bleeding complications: with Spinraza treatment, there was an increased risk of developing a low platelet count (thrombocytopenia) and abnormalities in blood clotting. Your doctor will check your blood before starting Spinraza and prior to each treatment. Tell your doctor right away if you have any unusual bleeding, such as nosebleeds, or bruising under your skin.

Kidney toxicity: kidney toxicity, including a potentially fatal condition called glomerulonephritis, has been observed after Spinraza treatment. Your doctor will monitor your kidneys by getting a spot urine protein test before initiating therapy and prior to each treatment. Tell your healthcare provider right away right away if you have any of the following symptoms of kidney dysfunction:

  • swelling of face, ankles, hands, or feet
  • fatigue
  • paleness of skin
  • decreased urination
  • shortness of breath
  • change in blood pressures

Spinraza Food Interactions

No known food interactions

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of Spinraza, there are no specific foods that you must exclude from your diet when receiving this medication. 

Inform MD

Before taking Spinraza, tell your doctor about all of your medical conditions.

Especially tell your doctor if you are allergic to Spinraza or to any of its ingredients.

Tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements. 

Spinraza and Pregnancy

Tell your doctor if you are pregnant or plan to become pregnant. 

There are no well-controlled studies that have been done in pregnant women. Spinraza should be used during pregnancy only if the possible benefit outweighs the possible risk to the unborn baby.

Spinraza and Lactation

Tell your doctor if you are breastfeeding or plan to breastfeed.

It is not known if Spinraza crosses into human milk. Because many medications can cross into human milk and because of the possibility for adverse reactions in nursing infants with use of this medication, a choice should be made whether to stop nursing or stop the use of this medication. Your doctor and you will decide if the benefits outweigh the risk of using Spinraza.

Spinraza Usage

Spinraza is given through a procedure called intrathecal injection by a trained healthcare provider. This delivers medication through the lower back via a lumbar puncture targeting the central nervous system.

Spinraza Dosage

The recommended dose of Spinraza is 12mg (5mL) per administration by intrathecal injection. 

Spinraza treatment is initiated as follows:

  • Start by giving four separate loading doses
  • The first three loading doses should be administered at 14-day intervals
  • The fourth loading dose should be administered 30 days after the third loading dose
  • Then, a maintenance dose should be administered once every four months thereafter

Spinraza Overdose

Spinraza is usually administered by a healthcare provider in a medical setting making it unlikely for an overdose to occur. However, if overdose is suspected, seek emergency medical attention. 


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