“The Remarkable Life of Ibelin” Review by Hawken Miller, Writer and Advocate When Mats Steen, a young Norwegian man, passed away from Duchenne muscular dystrophy at 25, his parents Robert […]
By Debra Miller, Founder and CEO of CureDuchenne I’m honored to share two transformative events that embody both the resilience of the human spirit and the strength of our community: […]
Read a message from our founder and CEO The recently released award-winning Netflix documentary “The Remarkable Life of Ibelin,”* highlights the life of Mats Steen, a Norwegian World of Warcraft […]
CureDuchenne welcomes the recent initiative the Food and Drug Administration (FDA) has taken to improve efficiencies in drug development, manufacturing, and the review process for new drug applications that incorporate […]
In a promising development, the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (ACHNDC) has advanced the nomination of Duchenne muscular dystrophy (DMD) for inclusion in the Recommended Uniform Screening Panel (RUSP), a list of disorders that are screened for at birth.
We at CureDuchenne are excited to acknowledge the recent announcement by the United States Food and Drug Administration (FDA) about the creation of the “Rare Disease Innovation Hub.” This new […]
A Father Rises in Support of His Son: On this powerful episode of the “In Sickness” podcast, dad, Josh Argall opens up about the emotional rollercoaster following his son Devin’s […]
See the previous update from June 22, 2023 here FDA Grants Traditional Approval for Ambulatory Individuals and Accelerated Approval for Non-Ambulatory Individuals to Sarepta Therapeutics’ ELEVIDYS Gene Therapy for Duchenne […]
As we prepare to welcome families to the CureDuchenne FUTURES Annual Conference, scientific leaders, executives from nearly every pharmaceutical and biotech company focused on developing therapies for Duchenne and Becker […]
