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 one of the first to get a special treatment that gave her a new chance at life. Mayo Clinic’s genetics team, led by Dr. Whitney Thompson and Dr. Laura Lambert, made this breakthrough by using existing drugs in new ways!
What started as a life-saving solution for Jorie has now grown into a strong 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.
