Molecular Genetics and Metabolism: Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic Mucopolysaccharidoses
On February 21, 2024, a public workshop was convened by the Reagan-Udall Foundation for the Food and Drug Administration (FDA) to explore primary disease activity biomarkers in rare genetic diseases using cerebrospinal fluid (CSF) heparan sulfate (HS) as a case study for a biomarker to support accelerated approval of new therapeutics for neuronopathic mucopolysaccharidoses (MPS) disorders.