In this episode, we speak with Irena Muffels and Eva Morava about their recent review, The Therapeutic Future for Congenital Disorders of Glycosylation. We explore the three pillars they propose for advancing CDG treatment: improving disease models, enhancing clinical trial readiness, and finding scalable strategies that move us beyond one-gene-at-a-time approaches. From patient-derived brain organoids to AI-powered outcome assessments and the promise of basket trials, this conversation is a sweeping tour through the innovations shaping the future of CDG care.

The Therapeutic Future for Congenital Disorders of Glycosylation

I. J. J. Muffels, T. Kozicz, E. O. Perlstein, E. Morava

https://doi.org/10.1002/jimd.70011