28% of Families:
have at least one child with a special healthcare need. Many of those are from genetic conditions. Our mission is to fund, inspire and accelerate n=1 therapies for children with rare diseases - because every child deserves a chance to rewrite what is possible.
Miracles Can Happen
Jorie, a brave child, was among the very first to receive a special, personalized treatment that gave her a second chance at life! Mayo Clinic's incredible genetics and research team, led by Dr. Whitney Thompson and Dr. Laura Lambert, achieved this breakthrough through drug repurposing.
What began as a unique, life-changing solution for one child has now blossomed into a powerful movement in personalized medicine!
The Jorie Effect Is:
Science in Motion
Hope with Wings
A New Model
Family First
Our vision:
To create a future where no child is left behind because their disease is too rare.
A world where the rarest lives spark the boldest breakthroughs.
Our mission.
To fund, inspire, and accelerate n=1 gene therapies for children with ultra-rare diseases— because every child deserves a chance to rewrite what’s possible.
Our Scientific Roots:
Jorie’s therapy was developed under the guidance of:
Dr. Whitney Thompson, M.D. M.Phil, Neonatology & Medical Geneticist Fellow Physician Mayo Clinic
A leading expert in pediatric genetic disorders with a deep commitment to translational care for rare disease patients.
Dr. Laura Lambert, Ph.D., Research Geneticist
Mayo Clinic
A driving force in developing and validating gene therapies for single-patient applications, with a focus on restoring protein function at the molecular level.
Their innovative work underscores the power of academic and clinical teams in advancing precision medicine for the rarest of patients.
