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Personalized Medicine Initiatives

Offer Hope to Acutely Ill Children

At Nicklaus Children’s Hospital

Nicklaus Children’s Hos - in less than a week. A con-
pital, in collaboration with firmed diagnosis can be vital
Rady Children’s Institute in the first days of life to direct
for Genomic Medicine and appropriate, and sometimes
Sanford Health, has life-saving, clinical care.
launched two genomic For diagnostic odyssey
medicine research proto- patients, WGS is used to pro-
cols to more effectively vide long-sought answers for
diagnose and treat children families in search of a diagno- is proud to honor
with unexplained illnesses. sis for a chronically ill child.
The three participating Often these children have vis-
entities have a shared com- ited multiple specialists and
mitment to clinical innova- undergone myriad tests and
tion and research collabo- Dr. Jennifer McCafferty procedures in their young
ration that enhance patient lives. Whole genome sequenc-
care, decrease healthcare costs and further ing can unlock answers for these families.
the scientific understanding of rare genetic Both efforts are part of the Personalized
diseases in children. Nicklaus Children’s Medicine Initiative at Nicklaus Children’s
Hospital and San Diego-based Rady Hospital led by Jennifer McCafferty, Ph.D.,
Children’s Institute, are part of the Sanford director of Nicklaus Children’s Research
Children’s Genomic Medicine Consor - Institute and Personalized Medicine
tium, which is committed to advancing Initiative.
children’s medicine through integration of “We are thrilled to be at the leading edge
genomics into pediatric care. Sanford of genomic medicine in pediatrics. This
Health is an integrated health system head- program is a great example of how
quartered in the Dakotas. Nicklaus Children’s partners on discovery
The goal of genomic medicine is to and delivers on clinical impact for children
develop a precision care plan for each child and families in South Florida,” said Dr.
based on determining an underlying McCafferty. “Genomic medicine offers
genetic diagnosis. The collaboration hope to parents of sick children who have
between Nicklaus Children’s and Rady not yet received a diagnosis.” Elizabeth Dudek
Children’s Institute will offer whole Both the diagnostic odyssey and inten-
genome sequencing (WGS) to children in sive care unit rWGS efforts are in the with the Claude Pepper Lifetime Achievement Award
two settings: research phase and not yet offered as a for her extraordinary contributions to health care and
• Acutely ill patients with undetermined standard of care. Physicians at Nicklaus
illnesses in Nicklaus Children’s three Children’s Hospital are able to refer poten- championing causes on behalf of the elderly.
intensive care units (neonatal, pediatric tial candidates to the research team for
and cardiac). These children will be eligi- possible enrollment in these initiatives. Please join us in congratulating Elizabeth Dudek,
ble for rapid whole genome sequencing Since July 2016, Rady Children’s
(rWGS) testing to provide the fastest pos- Institute for Genomic Medicine has per- Director of Health Care Affairs for Greenberg Traurig, P.A.,
sible results. formed rapid whole genome sequencing and former Secretary of the Agency for
• Children with chronic undiagnosed for more than 431 acutely ill children Health Care Administration.
medical conditions. These children, often enrolled in its research studies. As of
described as being on a “diagnostic April 9, approximately one-third of
odyssey,” have typically visited multiple patients sequenced have received a
specialists in search of a definitive diagno- genomic diagnosis with 63 percent of Congratulations to all the
sis to support treatment. those benefitting from an immediate
Through the collaboration, Nicklaus change in clinical care. Claude Pepper Awards Honorees!
Children’s will send patient blood samples “The information we receive from
to Rady Children’s Institute for Genomic whole genome sequencing is helping Lifetime Achievement Public Service
Medicine in San Diego. Whole genome medical teams make treatment decisions, Elizabeth Dudek, Honorable Esteban Bovo, Jr.
sequencing, bioinformatics analysis, and providing hope to these children and Director of Health Care Affairs Commissioner, District 13
initial clinical interpretation will occur in families,” said Stephen Kingsmore, M.D., Greenberg Traurig, P.A. Miami-Dade County
San Diego and the experts at Nicklaus DSc, president and CEO of Rady
Children’s will use the results to care for Children’s Institute for Genomic Medicine. Corporate Service - Healthcare Philanthropic Service
children in Miami. “This is the beginning of our long-term Penny S. Shaffer, Ph.D. The José Milton Foundation
For acutely ill newborns and children, vision to save babies with rare, life-threat-
rWGS, a relatively new test, can provide ening diseases by making genomic testing Market President - South Florida Cecil, Frank, Joseph & Ana Milton
much faster diagnoses than traditional available to NICUs, PICUs and CICUs Florida Blue
genome testing, mapping a whole genome nationwide.” Thelma Gibson
Corporate Service Community Service
Bacardi, U.S.A. Juan and Maria Del Busto
Eduardo Cutillas The Del Busto Family Foundation
Education / Advocacy Community Builder
Subscribe to... Dr. Mark B. Rosenberg, The Honorable Keon Hardemon

Commissioner – District 5
President, Florida
City of Miami
International University
SOUTH FLORIDA HOSPITAL NEWS &
Thursday, May 17, 2018
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