The N-of-1+ initiative is focused on the development of antisense oligonucleotide (ASO) therapeutics for patients with a serious, life-threatening rare disease where there are fewer than ~100 known patients worldwide.
The rapid progress in drug development for n=1 and ultra-rare diseases raises multiple complex issues. The OTS Rare Disease Task Force was created to help address some of these issues.
As the main non-profit society in this field, we seek to help in three ways:
- Formulating guidelines to help avoid wasted effort and dangerous projects
- Helping to streamline the process of drug development and approval by connecting patients/families/physicians with appropriate resources
- Helping to clarify expectations, guidelines, and protocols ideally in dialogue with the regulatory agencies.
The OTS is partnering with the newly formed N=1 Collaborative to organize workshops and advance our efforts for this initiative.
In The News
They Created a Drug for Susannah. What About Millions of Other Patients?
By Erika Check Hayden (New York Times) Photographs by Brittainy Newman Scientists have made rapid progress in customizing drugs for ultrarare diseases. The hard part now is making such treatments on a large scale. Susannah Rosen, 8, spent much of her childhood in hospitals in New York City as ...
