Patient-Funded Research For Rare Brain Disorder At Kennedy K

|Feb 28|magazine8 min read

BALTIMORE, Feb. 28, 2020 /PRNewswire/ -- In honor of International Rare Disease Day, a young rare disease advocate with a leukodystrophy, Ellie McGinn, presented her doctors at Kennedy Krieger Institute's Moser Center for Leukodystrophies with $450,000, bringing the total she and her family raised for research of the rare brain disease, Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation (LBSL), to $1.6 million in four years.

McGinn is one of only 100 patients worldwide with LBSL, a progressive condition impacting the brain and spinal cord of children. Through A Cure for Ellie, a non-profit dedicated to raising awareness and funds for LBSL research, McGinn and her family raised funds through events, silent auctions and social media since 2016, when the Center began researching LBSL.

S. Ali Fatemi, MD, chief medical officer at Kennedy Krieger, leads LBSL research at the Center where molecular disease mechanisms are studied, new therapeutic approaches identified, innovative clinical research projects leveraging wearable technologies are conducted; and machine learning tools advanced to better understand the clinical course of this rare disorder. The funds from A Cure for Ellie allow the Center to train aspiring researchers and physicians to become experts in LBSL. It is through this same funding that the Center's team is rapidly pushing forward toward the development of new therapies with the hope that within the next five years, the Center will commence clinical trials. 

About Kennedy Krieger Institute:
Kennedy Krieger Institute, an internationally known, non-profit organization located in the greater Baltimore/Washington, D.C. region, transforms the lives of more than 25,000 individuals a year through inpatient and outpatient medical, behavioral health and wellness therapies, home and community services, school-based programs, training and education for professionals and advocacy. Kennedy Krieger provides a wide range of services for children, adolescents and adults with diseases, disorders or injuries that impact the nervous system, ranging from mild to severe. The Institute is home to a team of investigators who contribute to the understanding of how disorders develop, while at the same time pioneer new interventions and methods of early diagnosis, prevention and treatment. Visit KennedyKrieger.org to support or for more information about Kennedy Krieger. 

About A Cure for Ellie 
"A Cure for Ellie" is a non-profit organization dedicated to raising awareness and funds for research into LBSL. Founded in 2013, the organization is named after a young girl in Arlington, Virginia named Ellie McGinn. At around age two-and-a-half, Ellie began showing classic symptoms of LBSL – leg spasms, poor balance, falling and hand tremors.  At age three-and-a-half she was finally diagnosed. Ellie and her parents found help and hope at the Kennedy Krieger Institute in Baltimore, Maryland where research is currently underway to test and develop novel drug therapies that will hopefully lead to a cure. Ellie's story was profiled nationally on NBC's Today Show by Kathy Lee Gifford and Hoda Kotb in 2017 soon after her ninth birthday.

MEDIA CONTACTS:   
A CURE FOR ELLIE
(202) 487-4235 

Grace Clark
PROFILES for KENNEDY KRIEGER INSTITUTE
410-243-3790

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SOURCE Kennedy Krieger Institute