At a symposium on rare diseases, smiles were in abundance

| Written by Scott LaFee
Malin Burnham, Debra Turner, Hudson Freeze and David Brenner
left to right: Malin Burnham, Sanford Burnham Prebys trustee; philanthropist Debra Turner; Hudson Freeze, Ph.D., director of the Sanford Children’s Health Research Center; and David Brenner, M.D., president and CEO of Sanford Burnham Prebys.

Since 2010, we have organized an annual scientific and family conference at Sanford Burnham Prebys that covers multiple rare diseases.

In alternate years, this meeting has focused on Congenital Disorders of Glycosylation (CDG).

Our 2024 meeting this month was a cause for celebration. Partnering with the family support and information group, CDG CARE, the Sanford Children’s Health Research Center and sponsors invited scientists, families and physicians to share their stories – some technical, some heart-breaking, some updates of ongoing therapies and some describing new developments.

Five years ago, there were no therapies; now seven are moving into patients. Celebration indeed.

Our keynote speaker, Joni Rutter, Ph.D., director of the National Center for Advancing Translational Sciences, part of the National Institutes of Health, commented on our event:  “Meetings that engage clinicians, scientists, advocates and families equally should be the standard. (Our) approach is a model of collaboration and impact."

In this CDG CARE video of this month’s gathering, you can see the joy, optimism and hope that inspires and helps carry us all. Thanks to everyone for their support. It makes those smiles real.

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