Who: 126 individuals ages 2-9 years with later-onset SMA
Study time: 15 months
Primary outcome: Changes in motor function measured on the HFMSE
Secondary outcome: Changes in upper limb function measured on the RULM and percentage of individuals who had a clinically meaningful improvement of 3 or more points from baseline in HFMSE score
Safety: The most common side effects were:
People on SPINRAZA had a 3.9-point improvement in overall motor function versus a 1.0-point decline in the control group.
Who: 28 individuals ages 2-16 years with later-onset SMA: 11 with Type 2 and 17 with Type 3
Study time: 3 years
Primary outcome: These supportive studies were designed to determine the safety of SPINRAZA
Other outcomes: The safety and longer-term effects of SPINRAZA on overall motor function, upper limb function and walking ability were also studied
Limitations: The dosing was different than the approved SPINRAZA schedule and these studies had no controls
Safety: Side effects were consistent with those reported in the pivotal trials
Who: 139 adults ages 16-65 years with later-onset SMA: 2 with Type 1, 47 with Type 2, 89 with Type 3, and 1 with Type 4
Study time: 14 months
Primary outcome: Changes in motor function at 6, 10, and 14 months, measured on HFMSE
Secondary outcomes:
Limitations
Safety: Most of the side effects were consistent with those in the SPINRAZA pivotal trials. The most common side effects in the trial were headache, back pain, and nausea. Others reported were:
Who: 116 adults ages 18-72 years with later-onset SMA; 13 with Type 2 and 103 with Type 3
Study time: 14 months
Primary outcomes: Changes in overall motor function measured on HFMSE, changes in upper limb function measured on RULM, and changes in walking ability measured on 6MWT
Limitations:
Safety: No new side effects were identified in this study; those that were reported were consistent with the safety profile of SPINRAZA.
The most common side effects were headache and back pain. Additionally:
“For me, it’s always been mind over matter; focusing on what you can do, and working around what you can’t.”