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Children's Hospital of Pittsburgh Foundation has received two grants, totaling $2.5 million, from The Legacy of Angels Foundation to support the diagnosis, treatment, and research of rare and devastating neurological diseases.
The funding will support the ongoing work of the Program for the Study of Neurodevelopment in Rare Disorders (NDRD) at Children’s Hospital of Pittsburgh of UPMC, a part of the recently established Center for Rare Disease Therapy.
Maria Luisa Escolar, MD, MS established the NDRD to help children and their families understand the impact of rare, inherited neurological diseases on child development. Started in 2000, the program focuses on genetic neurodegenerative diseases, such as Krabbe disease, an inherited condition caused by a faulty gene, and other leukodystrophies.
“Because these diseases are so rare, most physicians may see at most one person in their lifetime,” said Dr. Escolar, also associate professor, Pediatrics, University of Pittsburgh School of Medicine. “Through our program, we have seen more than 700 patients with rare disorders, performed more than 2,000 evaluations, and developed crucial information about these diseases that is unique in the world.”
The Legacy of Angels Foundation was founded in 2008 by Sue and Paul Rosenau to fund curative research into Krabbe disease. In 2003, the Rosenaus’ lives changed when they lost their granddaughter, Makayla, to the disease. Five years to the day later, the Rosenaus’ lives changed again when they won the Powerball. After winning the $180 million jackpot, the family established the Legacy of Angels Foundation in honor of Makayla and they remain devoted to finding improved treatments and research for Krabbe disease as well as cystic fibrosis.
Since Dr. Escolar’s arrival at Children’s Hospital in 2011, The Legacy of Angels Foundation has donated more than $5 million in support of her work. The two most recent grants received will help support the area of gene therapy, and also care of patients with Krabbe disease, and Krabbe research at the NDRD.
“We are beyond grateful for the Legacy of Angels Foundation and its continued generosity to help support Krabbe research, treatment development, and clinical services for families,” Dr. Escolar said. “Without this type of help, we would not be able to achieve our goal of improving the quality of life for children with rare disorders and help them develop to their full potential.”
“We started this Foundation to honor Makayla with the primary goal to further educate and enhance treatments and cures for Krabbe disease,” said Paul Rosenau, co-founder, Legacy of Angels Foundation. “Being given the chance to support Dr. Escolar and the NDRD, we know that families will have access to the most advanced clinical and research treatments for their child’s disease and the funding will continue to help other patients in the future.”
For more information on Dr. Escolar and the Program for the Study of Neurodevelopment in Rare Disorders, visit www.chp.edu/our-services/rare-disease-therapy.
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