N1C Preclinical Workgroup: Preclinical Modeling Systems

N1C Preclinical Workgroup: Ordering ASO Checklist

N1C Preclinical Workgroup: Valuable Tools for the Preclinical Stage of Developing Potential Therapeutics

Community Job Openings

N1C Patient Identification Workgroup: Compilation of Useful Tools

Community Events

‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine

The Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study

All of Us and the Promise of Precision Medicine:

MAKING GENETIC THERAPIES AFFORDABLE AND ACCESSIBLE

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs

Illustrated Example for n=1 Clinical Protocol

Illustrated Example for n=1 Consent Form

Clinical Recommendations Draft Guidance

CMC Recommendations Draft Guidance

Nonclinical Testing Draft Guidance

Administrative and Procedural Draft Guidance