Leveraging Connections & Technology For Rapid Clinical Material Delivery

For families facing a devastating diagnosis of an ultra-rare disease, time is the greatest challenge. In Part Three of this four-part series, we continue the remarkable journey of the Krueger family, whose daughter Elly was diagnosed with NEDAMSS—an ultra-rare, progressive neurodegenerative disorder—at just eight months old. After quickly assembling a team of experts, the Kruegers selected a manufacturing partner to produce a novel gene replacement therapy within just months of diagnosis.

This installment highlights the critical role of selecting the right CDMO, how Andelyn Biosciences AAV Curator® platform helped accelerate production, and how collaboration between scientists, service providers, and the family made it possible to overcome regulatory and logistical hurdles. It also explores how Andelyn supported Elly’s Team Foundation and contributed to raising awareness and funding for ultra-rare diseases.

Read Part Three of this series to learn how coordinated action, trust, and determination enabled a life-saving breakthrough in just 14 months.