Creating a
community
We are bringing together the biotech and pharmaceutical industries, patient advocates and foundations, expert physicians and researchers, organizations that support our discovery, development, manufacturing and clinical management efforts. Together we can help change the world one nano-rare patient at a time.
Sharing
experiences
We provide a platform for our nano-rare patients to share their experiences and lessons learned to the broader community. We welcome all our nano-rare family to participate.
Working with the
community
We believe that it is important to drive genomic sequencing into newborn evaluation protocols and are committed to working with our partners to support legislative and regulatory agendas.
Teaching the
science
We are recording an Intro to Medical Science series that provides insight into the world of drug discovery and development, how drugs are chemicals, how organs in the body work and become diseased and many more topics that provide the educational foundation for nano-rare patients to better understand their disease, their healthcare choices and the resources available.
Listen. Learn. Share.
Want to learn more?
Check out our podcast series!
New episodes added every other Wednesday.
Also available on: 
Please join us on a Corps of Discovery of the
Mind and Heart!
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.
Follow us on social for updates on our latest efforts
