Where one life inspires a ripple of discovery. Change a life today.
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 became one of the first patients to receive personalized therapy via drug repurposing that restored her levels to wild-type. Her groundbreaking treatment was developed and administered by Mayo’s genetics and research team, lead by Dr. Whitney Thompson, M.D M.Phil and Dr. Laura Lambert, P.H.D. What began as a single patient - n=1 - is now a movement.
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, Medical Geneticist, 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.
