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Nusinersen is available as a 12 mg/5 mL (2.4 mg/mL) single-dose vial.

Nusinersen is an injectable medication used to treat spinal muscular atrophy (SMA) in children and adults.

Nusinersen is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide. People with spinal muscular atrophy (SMA) don’t have enough of a protein called survival motor neuron (SMN), which is needed for your nerves and muscles to function properly. Nusinersen is an antisense oligonucleotide that helps the body make more SMN proteins to treat people with SMA.

The typical dose is 12 mg (5 mL) injected by intrathecal route (into the spinal canal). Nusinersen is injected as four loading doses. The first three doses are given every 2 weeks. The fourth dose given 1 month after the third dose. After all loading doses are complete, you’ll continue on a maintenance dose given once every 4 months.

Nusinersen is a safe and effective treatment when used for FDA licensed indications. However, like all medications, they may give you unwanted side effects. You should always discuss potential side effects with your physician to ensure the medication is suitable and right for you.

Common side effects of Nusinersen:

• Lower respiratory infection
• Constipation
• Urinary tract infection
• Stuffy nose
• Ear infection
• Gas
• Weight loss
• Fever
• Headache
• Vomiting
• Back pain
• Nosebleed
• Falls

Serious side effects are rare with Nusinersen. Contact your healthcare provider immediately if you experience any of the following:

• Serious bleeds: bleeding that’s difficult to stop, bloody or black-colored stool, vomiting or coughing up blood, dizziness, pale skin
• Kidney damage: low urine amount, swelling in legs or feet, shortness of breath, tiredness, confusion, nausea, weakness, irregular heartbeat, headache, or itchiness
• Atelectasis (collapsed lung): trouble breathing, fast heart rate, cough, chest pain, or skin and lips turning blue

There are no major drug interactions listed for this medication. However, always let your physician and pharmacist know about any other medications or supplements (including prescribed and over-the-counter medications, vitamins, and dietary or herbal supplements) that you are currently taking.

Various FDA-approved generic manufacturers

Based on animal data, Nusinersen may cause fetal harm. Talk to your doctor if you are already pregnant or if you have plans on becoming pregnant. They will recommend what is best for you.

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