Study: Phase 3, multicenter, randomized (2:1), double-blind, sham procedure–controlled
Treatment duration: 15 months
Participants: 126 patients with later-onset SMA aged 2 to 9 years at screening
Primary endpoint: Change in motor function as measured by HFMSE
Secondary endpoint: Change in upper limb function as measured by RULM
Study limitations: Differences in dosing compared to the approved SPINRAZA schedule
Safety: The most common side effects were fever (43%), headache (29%), vomiting (29%), and back pain (25%)
HFMSE=Hammersmith Functional Motor Scale—Expanded.
Motor function began to steadily improve in just 6 months compared to untreated group.3
RULM=The Revised Upper Limb Module evaluates limb function and strength in individuals with later-onset SMA who are unable to walk. It is scored from 0-37 points, with higher scores indicating better function.
Study: Phase 1b/2a, open-label, multiple-dose, dose-escalation integrated analysis of CS2 and CS12 studies
Treatment duration: ~3 years (38 months)
Participants: 28 ambulatory and nonambulatory patients with SMA aged 2 to 16 years at time of first dose
Primary endpoint: Safety of SPINRAZA
Study limitations: Differences in dosing compared to the approved SPINRAZA schedule and no control group
Safety: Adverse reactions were similar to those reported in the pivotal trials
HFMSE was developed specifically for SMA and is a widely used and well-validated scale for measuring clinical benefit in later-onset SMA.6,7
ULM=The Upper Limb Module evaluates limb function and strength in individuals with later-onset SMA who are unable to walk. It is scored from 0-18 points, with higher scores indicating better function.
SPINRAZA may help patients gain or regain the ability to walk.4
“My belief is that anyone can do anything. It doesn’t matter if you’re in a wheelchair, it doesn’t take much strength to make
a big impact.”