The Continuation Of A Journey

Crafting a clinical-grade gene replacement therapy for Elly Krueger in just 14 months was an incredible feat. This remarkable achievement was possible due to the constant coordination and management of many different groups and individuals by Elly's parents, Dan and Michelle.

The success was a true team effort:

• Dr. Zachary Grinspan's support, expertise, and guidance were crucial. He was on call on Thanksgiving 2023 and played a key role in the Kruegers' efforts to develop the treatment.
• Dr. Kathrin Meyers' team provided the initial evidence that gene therapy could help Elly. Her encouragement and network were invaluable.
• The University of Missouri team demonstrated incredible dedication, completing proof-of-concept and safety studies on a highly accelerated timeline.
• The team at Andelyn Biosciences was willing to take calls at any time, which allowed the investigational new drug (IND) application to the Food and Drug Administration to proceed smoothly.

This collaborative effort, involving so many different people and organizations, highlights the immense challenges families face when navigating a complex diagnosis like NEDAMSS.