Accelerating Development Of Gene Replacement Therapy To Treat NEDAMSS

In the face of a devastating diagnosis, Michelle and Dan Krueger refused to accept the status quo. In just 14 months, they led a remarkable effort to develop a personalized gene replacement therapy for their daughter, Elly, who was diagnosed with NEDAMSS. This progressive, ultra-rare neurodegenerative disorder affects roughly 150 children worldwide. In Part TWO of this compelling series, the focus shifts from Elly’s diagnosis to the science behind the disease and the rapid, coordinated effort required to create a viable treatment.

Highlighting the groundbreaking work of Dr. Kathrin Meyer and her collaborators, who characterized the IRF2BPL mutation behind NEDAMSS and developed a gene replacement therapy based on an AAV9 vector. It explores how the Kruegers accelerated timelines by pursuing parallel preclinical and manufacturing work, leveraging trusted partners, and making bold but calculated decisions. Learn how an innovative delivery strategy using MRI modeling helped bring this treatment to life—and what the future might hold for Elly and others like her.