- An ultra-rare, fatal childhood condition exists with no effective treatment
- Condition affects 36 infants per year in the U.S.
- Regulatory and Quality Consulting consultants submit an Orphan Drug and Pre-IND/End of Phase 2 (EOP2) Briefing Document to FDA
- Secure meeting with the FDA
- FDA approves small clinical trial (25 patients)
- Orphan Designation granted
- Improved chances of therapy development