MAINTAINING MOTOR FUNCTION
Individuals with later-onset (Types
2 and 3) maintained various improvements in motor function
over nearly 3 years of treatment
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.
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
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
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:
Using a pencil
Picking up a coin and placing it in a cup
Pressing a button to turn on a lamp
Lifting a beverage can to drink
Lifting a weight and moving it from circle to circle on a preprinted paper
Removing a lid from a plastic container
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.
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
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.