OTS Rare Disease Task Force

 

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:

1. Formulating guidelines to help avoid wasted effort and dangerous projects
2. Helping to streamline the process of drug development and approval by connecting patients/families/physicians with appropriate resources
3. Helping to clarify expectations, guidelines, and protocols ideally in dialogue with the FDA

 

To learn more, click here to read our briefing document.

Rare Disease Briefing Document

If you are interested in an upcoming workshop on this topic, click here.

OTS Rare Disease Workshop