Gene Target
K1F1A
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ASO Strategy
Allele selective
PATIENT NL-00255
PATIENT NL-00037
Patient NL-00255
Treatment Start Date:
October 2022
Milestones
Standing up
For the first time, Susannah is able to stand on her own. Proud of herself, she playfully teaches her father ‘how to stand up’.
Playing basketball
Testing her motor control skills, Sus shoots a basketball with her brother.
Climbing Stairs
Climbing stairs involves coordinated muscle activation in the legs, hips, and core to propel the body upwards step-by-step, while maintaining balance and proper weight distribution, making it a demanding activity that tests multiple aspects of neuromuscular function.
Putting on shoes
For the first time, Susannah puts on her shoes by herself
Patient NL-00255
Treatment Start Date:
May 2024
Milestones
Counting numbers
Sloane is able to focus and count to 10
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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