Gene Target
SCN2A
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ASO Strategy
Allele selective
PATIENT NL-00001
PATIENT NL-00333
Patient NL-00001
Treatment Start Date:
March 2024
Milestones
Assisted Walk
Connor walks with the help of his mom, never being able to take more than a few steps on his own
“He’s literally walking”
For the first time, Connor is able to walk a short distance unassisted
Improved distance
Connor walks a significantly longer distance with improvement in balance
We cannot do
this alone
Together we are changing the world—
one patient at a time
We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.
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