Who: 25 infants aged 6 weeks and younger who had not yet shown symptoms of SMA
Study time: This supportive study is ongoing
Primary outcomes: Time to death or respiratory intervention†
Secondary outcomes: The effect SPINRAZA has on reaching WHO motor milestones
Limitations: Small number of participants. The study is open-label.
Safety: Consistent with the SPINRAZA prescribing information. After a follow-up of over 14 months to 5.7 years, 25/25 infants experienced any AE (mild, moderate, or severe), and no new safety concerns were identified
At the initial interim analysis performed after all infants had received SPINRAZA for at least 14 months (median, 25 months; range, 14 to 34 months), 100% (25/25) of infants were alive without the need for permanent ventilation‡, 100% (25/25) were sitting without support, 88% (22/25) were walking with assistance, and 77% (17/22) of infants were walking independently.
†Respiratory intervention is defined as ventilation for ≥6 hours/day continuously for ≥7 days, or tracheostomy.
‡Permanent ventilation is defined as ventilation for ≥16 hours/day for >21 days in absence of an acute reversible event, or tracheostomy.
The majority of presymptomatic infants achieved the following WHO motor milestones:
*At least half of patients were followed for at least 4.9 years.
88%22 out of 25
able to suck/
based on maximal HINE-1 score at the
day 778 visit
84%21 out of 25
92%23 out of 25 never needed
suction excess saliva or
based on caregiver assessment of swallowing difficulty during mealtime over a period of 7 days
§At least half of patients were followed for at least 3.8 years.
“For Camden to do what he is able
do, we are just so thankful.”