Hopefully Emma begins her Spinraza treatments on Tuesday, 10/31… Halloween.
The following is from the Biogen pamphlet, “Unlock Her Inner Star”.
SPINRAZA may help improve motor milestone development
Infants taking SPINRAZA showed significant motor milestone improvements early on (day 183) in comparison with untreated infants.
ENDEAR was a well-controlled, 13-month, phase 3 clinical study with 2:1 randomization, meaning two-thirds of the individuals received SPINRAZA and one-third was untreated. The study was ended early as a result of clinically meaningful benefits seen in the planned interim analysis at day 183. All individuals were then moved into open-label extensions for further clinical study.
The interim analysis assessed the proportion of “responders,” or individuals with improvement in motor milestones, according to Section 2 of Hammersmith Infant Neurological Examination (HINE).
HINE motor milestone responders at day 183
SPINRAZA (n=52) UNTREATED (n=30)
40% Infants with milestone 0% Infants with milestone
improvements improvements
Ability to kick: 27% Ability to kick: 0%
Head control: 33% Head control: 0%
Rolling: 35% Rolling: 0%
Sitting: 25% Sitting: 0%
Crawling: 8% Crawling: 0%
Standing: 8% Standing: 0%
Walking: 0%* Walking: 0%*
* Individuals in this study were not of an age at which a healthy infant would typically be expected to walk.
EmmaStrong2017 
This sounds like an amazing new drug. Fingers crossed for Tuesday!!
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