News and media
Select a category
Among the three patients treated this fall is Susannah, a beautiful 8 year old girl who suffers from a neurodegenerative nano-rare condition. Susannah is receiving a personalized, experimental ASO discovered and developed for her unique mutation.
We are continuously developing new partnerships, new treatments, and solutions to those with nano-rare diseases. Contact us to learn more about our organization.
We cannot do
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.
Follow us on social for updates on our latest efforts