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MAINTAINING MOTOR FUNCTION

Individuals with later-onset (Types 2 and 3) maintained various improvements in motor function over nearly 3 years of treatment

Claire
age 19

Later-onset (Type 2) SMA
treated with SPINRAZA

Type 2 SPINRAZA® (nusinersen) patient motor function

The long-term effects of SPINRAZA were evaluated over a 3-year period (1050 days) in 28 individuals with later-onset (Types 2 and 3) SMA ranging from 2 to 16 years old.

Individuals maintained improvements in motor function that were measured by assessments that included the Hammersmith Functional Motor Scale—Expanded (HFMSE), Upper Limb Module (ULM), and 6-Minute Walk Test (6MWT).

This was an open-label study and all individuals received SPINRAZA. Limitations included differences in dosing compared with the approved regimen and lack of an untreated group.

Improvements in motor function seen over 1050 days

Regardless of their level of function before starting treatment (baseline), individuals maintained significant increases in their HFMSE scores over nearly 3 years of treatment.

Hammersmith Functional Motor Scale – Expanded for Type 2 Type 3 SMA motor function

The Hammersmith Functional Motor Scale—Expanded (HFMSE)

The HFMSE assesses motor function of individuals older than 24 months of age.
It measures 33 items grouped into 7 categories:

  • Each item is scored from 0 to 2
  • The maximum score is 66
measuring motor function for Type 2 Type 3 SMA patients

Without treatment, individuals with later-onset SMA typically lose motor function over time.

Improving upper limb function

An assessment called the Upper Limb Module (ULM) was used to evaluate limb function and strength in individuals with later-onset SMA who were unable to walk. The ULM is scored from 0 to 18 points, with higher scores indicating better function.

Over the course of 3 years (1050 days), individuals treated with SPINRAZA reached an average ULM score of 16.5, which is just 1.5 points below the maximum score of 18.

Improvements in ULM at day 1050

Increased upper limb function for Type 2 and Type 3 SMA patients

The Upper Limb Module (ULM)

The ULM includes 9 tasks that reflect activities of daily living and can typically be performed in a brief amount of time (5 to 10 minutes) using common equipment. Activities include:

Upper limb function for SMA patients using a pencil
PENCIL

Using a pencil

Upper limb function for SMA patients picking up a coin and placing it in a cup
COIN

Picking up a coin and placing it in a cup

Upper limb function for SMA patients pressing a button to turn on a lamp
PUSH BUTTON LIGHT

Pressing a button to turn on a lamp

Upper limb function for SMA patients drinking from a can
CAN

Lifting a beverage can to drink

Upper limb function for SMA patients lifting weight and moving it on paper
WEIGHTS

Lifting a weight and moving it from circle to circle on a preprinted paper

Upper limb function for SMA patients removing lid from plastic containe
LID

Removing a lid from a plastic container

Type 2 SMA patient holding a pen to write

One of my favorite activities is writing. I think a lot of people forget how physical writing is. The ability to hold a pen in a particular way that it needs to be held—to be able to write dark enough for you to actually be able to go back and read it.

My friends and I are spread out all across the country and even though we’re talking or texting to each other every day on the phone, we still love to write letters. Nobody writes letters anymore. I would never want to lose my ability to write.

—Claire

Improving walking ability

The 6-Minute Walk Test (6MWT) was used to evaluate the walking ability and endurance of ambulatory individuals with later-onset SMA. 100% of individuals treated with SPINRAZA (7/7) for at least 1050 days achieved a significant improvement in their ability to walk.

Improvements in 6MWT at day 1050

SPINRAZA® (nusinersen) patient walking distance
1 of 11 INDIVIDUALS

with Type 2 SMA gained the ability to walk independently

2 of 4 INDIVIDUALS

with Type 3 SMA regained the ability to walk independently


The achievement of this milestone in an individual with Type 2 SMA is inconsistent with the natural history of the disease, as those with Type 2 SMA are typically unable to walk independently.

The 6-Minute Walk Test (6MWT)

The 6MWT measures the distance an individual can walk in 6 minutes. Individuals are instructed to walk as far as possible along a 25-meter course and return in the opposite direction. The course is repeated as often as possible for 6 minutes.

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.