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UNLOCK THE POTENTIAL BENEFITS OF SPINRAZA

The SPINRAZA clinical study program is the largest in spinal muscular atrophy (SMA) to date

Claire
age 19

Later-onset (Type 2) SMA
treated with SPINRAZA

Type 2 Spinraza patient storie

The effectiveness of SPINRAZA was established in the pivotal ENDEAR study and supported by multiple clinical studies. Results were measured differently based on factors such as the age and physical ability of individuals in the clinical studies.

For individuals with infantile-onset (Type 1) and presymptomatic SMA in the ENDEAR and NURTURE studies, results included the achievement of developmental motor milestones.

The CS2/CS12 studies focused on individuals with later-onset (Types 2 and 3) SMA, evaluating improvements and maintenance of motor function over time, as well as specific motor skills—for example, walking and upper limb function.

Click through the tabs below for a closer look at the study results across a range of individuals with SMA.

INFANTILE-ONSET SMA

ENDEAR:
Pivotal controlled study
Age: 30-262 days

LATER-ONSET SMA

CS2/CS12:
Supportive, open-label study
Age: 2-16 years

PRESYMPTOMATIC SMA

NURTURE:
Supportive, open-label study
Age: 3-42 days

What are controlled and open-label studies?


Clinical studies are designed to determine whether a medication is effective and to monitor for any side effects. In a controlled study, there are treated and untreated groups, and the researchers and patients do not know who is receiving the drug. In an open-label study, both researchers and patients know what treatment is being given.

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.