Penn Med establishes program for rare disease

The London-based LouLou Foundation and the Orphan Disease Center of the Perelman School of Medicine have established a Program of Excellence to develop effective treatments for children with CDKL5, a rare X-chromosome-linked genetic disorder that causes severe neuro-developmental impair

The London-based LouLou Foundation and the Orphan Disease Center of the Perelman School of Medicine have established a Program of Excellence to develop effective treatments for children with CDKL5, a rare X-chromosome-linked genetic disorder that causes severe neuro-developmental impairment and early-onset, difficult-to-control seizures.

The LouLou Foundation will provide a three-year, renewable umbrella grant to fund all aspects of the program in order to help identify and fill gaps in basic research needed to develop medications and treatments for the disease, promote collaboration among CDKL5 investigators at leading other institutions, and engage with the biopharma industry.

“We are extremely grateful to LouLou Foundation for this grant,” says James M. Wilson, director of the Orphan Disease Center and a professor at Penn Medicine. “The marketplace provides few incentives for private-sector support of urgently needed research for orphan diseases such as CDKL5.”

Originally published on .