Qualifying Biomarkers to Support Rare Disease Regulatory Pathways
Case example: Heparan sulfate in neuronopathic lysosomal storage diseases

Public Workshop: In-Person and Virtual
February 21, 2024 | 10am-4pm (eastern)

On February 21 we convened "Qualifying Biomarkers to Support Rare Disease Regulatory Pathways," a day-long hybrid public workshop.

This workshop explored primary disease activity biomarkers in rare genetic diseases using heparan sulfate in neuronopathic mucopolysaccharidoses (MPS) as a case study for a biomarker to support accelerated approval. Featured perspectives included FDA representatives, patient advocates, researchers, and representatives from regulated industry. Scroll down to view the recording and workshop materials.