Toggle navigation
About The Disease
Elly’s Team
Letter To Community
Media
Contact Us
Donate
14 Months to Treatment: Breaking Records in Rare Disease Therapy Podcast
The Continuation of a Journey
Leveraging Connections & Technology for Rapid Clinical Material Delivery
Accelerating Development of Gene Replacement Therapy to Treat NEDAMSS
Realization of a Treatment through Grit, Commitment, Access, Resources, and Tremendous Collaborations
Andelyn Biosciences Applies the AAV Curator® Platform to Deliver Novel Gene Therapy for an Ultra-Rare NEDAMSS Disease Patient 14-Months after Diagnosis
Genezen Partners with Elly’s Team on Development for AAV Serotype 9 Gene Replacement Therapy for NEDAMSS; Genezen CTO Dr. Susan D’Costa to join nonprofit’s board
