Spinal muscular atrophy (SMA) is a rare disease that can affect one in 10,000 people. It is caused by a defective gene and has three different types: type I, type II, and type III. The most severe form of the disease is Type II spinal muscular atrophy (SMA). People with this condition have a shortened life expectancy and may lose the ability to walk as they get older.

Treatment for Spinal Muscular Atrophy usually involves physical therapy, speech therapy, adaptive training, and social support services. If you have been diagnosed with Spinal Musacter Atrophy, your doctor may prescribe Spinraza. This article explains how it works.

What is Spinraza?


Spinraza is a drug that is used to treat spinal muscular atrophy (SMA), a rare disease that affects the spinal cord and muscles. It is the first drug approved to treat all types of SMA. SMA is caused by a defect in a gene that leads to a breakdown of the spinal cord and muscles. Spinraza is a type of drug called an antisense oligonucleotide.

It is injected into the fluid around the spinal cord every four months. Spinraza is not a cure for SMA, but it can help reduce symptoms and increase mobility, muscle strength, and breathing. Spinraza does not work for everyone. You should not take this drug if you have an infection or you are unable to urinate.

How does Spinraza Work?

Spinraza is an injected drug designed to repair the gene defect associated with spinal muscular atrophy (SMA). SMA is a rare disease that causes the degeneration of motor neurons, which are responsible for muscle movement. The drug works by delivering a specific RNA molecule into the spinal cord cells that produce the defective gene, which is the root cause of SMA.

Specifically, Spinraza works by targeting the faulty RNA (the genetic material of the cell) and “silencing” it. In other words, it aims to interrupt the process that leads to the production of defective proteins. These defective proteins lead to the degeneration of the motor neurons that are responsible for muscle movement.

Side Effects of Spinraza

The most common side effects associated with Spinraza are mild to moderate infections that can occur at the site of the injection. Other side effects include trouble urinating, vomiting, nausea, headache, and joint pain. There have also been rare reports of spinal cord abnormalities and blood clots.

Cost of Spinraza

Spinraza is very expensive. The drug costs $750,000 per year per patient. Insurance may cover some of the costs. You should check with your insurance company to see if you qualify for benefits. Many people with SMA experience high medical expenses, especially as they get older and require more care. To help ease the financial burden of these expenses, the SMA Foundation offers a financial assistance program.

Conclusion: Who is Spinraza for?

Spinraza is approved to treat all types of SMA, including the most severe form, type II. It is not recommended for people with the milder type I form of SMA. Spinraza is administered via injection into the fluid around the spinal cord. Doctors recommend that you be treated at least one year after diagnosis. Spinraza can help reduce symptoms, increase mobility, muscle strength, and breathing. However, it is important to note that the drug is not a cure for SMA.