Some infants achieved the ability to sit without support
Evrysdi helped infants survive without permanent breathing support
Out of the infants taking Evrysdi
The infants in this part of the study ranged in age from 3 to 7 months at the start of treatment, but many were closer to 7 months.
Additional exploratory observations in eating and swallowing
This information is considered exploratory. This means it was not designed to show a treatment effect so conclusions cannot be drawn from these observational data.
Out of the infants taking the recommended dose of Evrysdi for 12 months:
- 88% were able to eat by mouth (15 out of 17 infants)†
- 88% were able to swallow (15 out of 17 infants)‡
*Permanent support was defined as having a
tracheostomy (a surgery where a tube is inserted in the front of the
throat into the windpipe) or more than 21 days of noninvasive
ventilation support (16 or more hours a day) or being intubated (a
procedure where a breathing tube is inserted down the throat and
into the windpipe) to help with breathing in the absence of an acute
†Two of these infants could eat some food by mouth, but also used a feeding tube.
‡One infant could not swallow at the start of the study.