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

Video Timeline

Clinical Data

Susannah’s Story

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

Clinical Data

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.

We need your support

Join us on our Corps of Discovery of the mind and heart. Help us bring hope and potential help to nano-rare patients today. For free, for life.

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