David Fajgenbaum, Associate Professor of Medicine in Translational Medicine and Human Genetics at University of Pennsylvania School of Medicine, shared a post on LinkedIn:
“We are proud to announce that Every Cure is launching a new drug repurposing program focused on DFMO for Bachmann–Bupp Syndrome, an ultra-rare condition that has been identified in only about 20 children worldwide.
Children with Bachmann–Bupp Syndrome often require feeding tubes, have limited motor control, and face significant daily challenges. The condition was first described by Dr. Bachmann and Dr. Bupp, who also pioneered the use of DFMO—a drug originally developed to treat African sleeping sickness—to target the underlying biology of this disease. In treated patients, DFMO has shown the potential to reverse key symptoms, including removing feeding tubes and helping children sit up and interact more fully with their families.
So far, approximately six children have been treated with DFMO, with meaningful improvement observed in five of them. This is exactly the kind of condition that is often overlooked because of its rarity and exactly why drug repurposing matters.
We are deeply grateful to Drs. Bachmann and Bupp for their groundbreaking work, and we are honored to partner with them to identify more patients, expand access to treatment, and help unlock better outcomes for children and families living with this devastating condition.
Read the full press release.”
More posts from David Fajgenbaum.