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THE SCIENCE
BEHIND SPINRAZA

SPINRAZA increases survival motor neuron (SMN) protein, addressing the underlying cause of motor neuron loss in spinal muscular atrophy (SMA)

Emma
age 7

Later-onset (Type 3) SMA
treated with SPINRAZA

Patient taking SPINRAZA swimming

Wondering how SPINRAZA works? Let Emma explain.

Individual with SMA

SMN1  GENE

Missing or mutated

SMN2  GENE

A “backup” to SMN1

In individuals with SMA, a mutated or deleted survival motor neuron 1 (SMN1) gene does not produce enough SMN protein to maintain the survival of motor neurons. These neurons control muscle activity by sending signals from the central nervous system (CNS).

Without a proper level of SMN protein, motor neurons in the spinal cord will be lost, preventing the body’s muscles from receiving signals from the brain. This can lead to progressive muscle weakness and wasting (atrophy).

Survival motor neuron 2 (SMN2) is a closely related “backup” gene that is not believed to be affected in SMA, but does not produce enough full length SMN protein on its own.

Individual taking SPINRAZA

SMN2  GENE

+

SPINRAZA

Increased SMN protein production

BY TARGETING THE SMN2  GENE, SPINRAZA HAS BEEN SHOWN TO INCREASE ITS PRODUCTION OF SMN PROTEIN

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 of 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.

Please see full Prescribing Information for additional Important Safety Information.

As a courtesy, our full Prescribing Information is also available en Español. For prescribing decisions, please refer to official approved labeling.

This information is not intended to replace discussions with your healthcare provider.


INDICATION

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