Kennedy Krieger Named Center of Excellence For its Treatment of Rare Pediatric Diseases

tags: Rett Syndrome and Related Disorders Clinic Center for Genetic Muscle Disorders Center for Leukodystrophies Latest News

BALTIMORE, November 4, 2021Kennedy Krieger Institute has been designated as a National Organization for Rare Disorders (NORD) Rare Disease Center of Excellence. The designation recognizes centers that are leaders in the diagnosis and care for people with rare diseases. The Institute receives the new designation in partnership with Johns Hopkins Medicine.

Kennedy Krieger treats more than 50 rare diseases in pediatric patients, including leukodystrophy, muscular dystrophy, Kabuki syndrome, Rett syndrome as well as other disorders that impact the nervous system. The Institute is home to several rare disease programs that also have been designated as Centers of Excellence by various organizations, such as the Center for Genetic Muscle Disorders, the Moser Center for Leukodystrophies, the Rett and Related Disorders Clinic, and the SYNGAP clinic.

Across the nation, 30 additional hospitals received NORD’s Center of Excellence designation. This network of centers will serve to expand access to care and research for rare diseases. A disease is considered rare in the U.S. when it affects fewer than 200,000 people at any given time; however, one in 10 Americans has a rare disease, which means an estimated 25-30 million Americans currently living with one of these conditions.

More than 90% of rare diseases lack an FDA-approved treatment, and establishing centers of excellence is one way to increase collaboration and better outcomes for patients with rare diseases, researchers agreed.

“Our clinicians at Kennedy Krieger look forward to this partnership with Johns Hopkins Genetic Medicine to further our efforts to care for patients with rare diseases,” said S. Ali Fatemi, MD, chief medical officer at Kennedy Krieger Institute and director of the Division of Neurogenetics and the Moser Center for Leukodystrophies. “Some of our patients have diseases that affect only 100 people in the world, so these partnerships and designations are instrumental to make progress toward determining effective treatments and improving children’s lives.”

At Johns Hopkins Medicine, the McKusick-Nathans Institute of Genetic Medicine | Department of Genetic Medicine houses several clinics and research centers dedicated to the discovery, research and treatment of rare diseases. One such center is Johns Hopkins Genomics, one of the world’s largest research genotyping and sequencing centers. Additionally, the Baylor-Hopkins Center for Mendelian Genomics, another such center, aims to sequence the genomes of people with rare diseases and their families to find the underlying genes and variants linked to the diseases.

“Centers and clinics like these enhance our ability to support the rare disease community,” says Ada Hamosh, M.D., clinical director of the department of genetic medicine at the Johns Hopkins University School of Medicine.

Established in 1983, NORD is a patient-focused organization that offers programs of education, advocacy, research and patient services to people with rare disorders.

About Kennedy Krieger Institute:

Kennedy Krieger Institute, an internationally known nonprofit 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 and 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 pioneering new interventions and methods of early diagnosis, prevention and treatment. Visit for more information about Kennedy Krieger.     


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