LouLou Foundation and the University Of Pennsylvania Create Program Of Excellence for Rare Genetic Disorder that Affects Children
CDKL5 causes severe neurodevelopmental impairment, seizures
5th February 2016 - London - The LouLou Foundation and the Orphan Disease Center of the Perelman School of Medicine at the University of Pennsylvania 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.
There are more than 1,200 documented cases of CDKL5 worldwide, with the number of identified patients increasing as genetic testing for the disorder becomes more common. The LouLou Foundation will provide a three-year renewable umbrella grant to fully 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 worldwide, and engage with the biopharma industry.
The focused grant program will welcome applications from academic labs and companies around the world and will be managed by a program director to be recruited specifically for this purpose. The size of the umbrella grant will depend on the number of grant applications ultimately awarded, but the partners are confident that the resources generously committed upfront by the LouLou Foundation, in combination with Penn’s ongoing investment in orphan disease research, will make a substantial positive impact on tackling CDKL5.
“We are extremely grateful to LouLou Foundation for this grant,” said James M. Wilson, MD, PhD, director of the Orphan Disease Center and a professor of Medicine. “The marketplace provides few incentives for private-sector support of urgently needed research for orphan diseases such as CDKL5. This program will promote cooperation and synergies with other leading academic medical centers and public and private institutions, all aimed at bringing the fruits of innovative research into the clinic as rapidly as possible.”
“Penn Medicine’s Orphan Disease Center has quickly established itself as an international leader in promoting effective partnerships to make inroads against orphan diseases through its unique model to fund the best science objectively and wherever it may be,” said a spokesperson for the LouLou Foundation. “We are delighted to begin this exciting collaboration with the center on behalf of all the children and families affected by this devastating disease.”