Story perspectives

First Patient Treated with ASO Therapy Shows Promising Progress

1/30/2026

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Summary

Story summary

In 2016, Susannah Rosen was diagnosed with KIF1A-Associated Neurological Disorder (KAND) due to developmental delays.
Her physician, Dr. Wendy Chung, advised her family to find a treatment within five years, prompting them to establish the KIF1A.org Foundation, which connects 700 families to support research.
The n-Lorem Foundation, founded by Dr. Stanley Crooke, developed antisense oligonucleotide (ASO) therapies for rare diseases.
Susannah became the first patient treated with ASO therapy, which targets her gene mutation and enables normal protein production.
After her second dose, her family noted positive changes in her behavior.

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Story summary

In 2016, Susannah Rosen was diagnosed with KIF1A-Associated Neurological Disorder (KAND) due to developmental delays.
Her physician, Dr. Wendy Chung, advised her family to find a treatment within five years, prompting them to establish the KIF1A.org Foundation, which connects 700 families to support research.
The n-Lorem Foundation, founded by Dr. Stanley Crooke, developed antisense oligonucleotide (ASO) therapies for rare diseases.
Susannah became the first patient treated with ASO therapy, which targets her gene mutation and enables normal protein production.
After her second dose, her family noted positive changes in her behavior.