Jill Weimer

Submitted photo

Sanford Health physicians and leaders were among the international experts presenting at the Fourth International Vatican Conference in Rome on April 26-28. The conference brings together leaders in health care, science and research as part of the Cura Foundation conference.  This is the second time Sanford physicians have presented their work at this conference.  Jill Weimer is the senior director of therapeutic development and associate scientist at Sanford Research.

Kealey Bultena

A treatment developed at Sanford Research is in clinical trial to help kids with a debilitating genetic condition. The FDA fast-tracked the study in part because the condition progresses quickly and children die.

Six patients are part of the Batten Disease trial. One family from the Midwest has a child treated.

Beth and Bryan live in Minnesota. At four years old, their son Blake struggled with fine motor skills. They incorporated occupational therapy. Then Blake needed speech therapy. Then his gross motor skills deteriorated, and he needed physical therapy.

Jill Weimer, Ph.D. recently was awarded $440,000 to support her research of a rare neurodegenerative disease called Batten Disease.  Weimer's lab is among only a few in the world studying the condition which primarily affects children that can cause seizures, blindness, motor and cognitive decline and premature death. Genetic mutations disrupt the ability of cells to dispose of waste and causes abnormal accumulation of proteins and lipids within nerve cells.  The grant funding will allow Weimer to screen several different treatment methods, which can include gene therapy or stem cells.