Mitchell and Friends Supports Promising Research at WashU

This month members of the Mitchell and Friends team visited the WashU Neuroscience Research Building in St. Louis to deliver another $50,000 donation to support research into Mitchell Syndrome and possible treatments for this ultra-rare disease.

That research is taking place at the Miller Lab under the direction of Dr. Timothy Miller and is being led by WashU scientist Dr. Zita Hubler. Dr. Hubler gave the team an update on what she is learning about how ACOX1 gain-of-function (Mitchell Syndrome) affects nerve cells. She also explained how cutting-edge antisense oligonucleotide (ASO) treatments might effectively target ACOX1 genetic mutations in those cells and so reduce the disease’s debilitating symptoms. This $50,000 donation will help pay for lab time and supplies as Dr. Hubler and her team continue their promising inquiry. With what they are learning about this disease, they hope to add to the growing body of knowledge about rare genetic neurological diseases and also apply for additional research grants. Ultimately, we all hope to help people affected by these illnesses live long and healthy lives.

This donation was made possible by the generosity of Mitchell and Friends supporters, and the proceeds of our 2024 Trivia Night. The research itself is made possible by the sacrifice and donation of Patient #1, Mitchell Herndon, who gave his body to WashU following his death, to support these efforts. Other patients have also donated bio-samples.

With their sacrifice—and with yours—we can defeat the rarest of rare diseases.