First Patient Enrolls in STOPSC Registry

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News From the LeBonheur Children's Medical Center

http://www.lebonheur.org/portal/site/lebonheur/

First Patient Enrolls in STOPSC Registry

On Monday, July 16, Sledge, a 10-year-old from Water Valley,

Miss., was the first patient to be enrolled in the STOPSC registry, a

multi-center database and DNA repository for patients with primary

sclerosing cholangitis (PSC). The registry is sponsored by the

Musette and , Jr. Foundation for the Study of Primary

Sclerosing Cholangitis, headquartered at Le Bonheur Children's

Medical Center.

PSC is a rare disease that causes the bile ducts inside and outside

of the liver to become scarred, narrowed and eventually blocked.

Often the only effective treatment for this debilitating disease is a

liver transplant. While children are waiting for a liver transplant,

they suffer with such symptoms as severe abdominal pain, jaundice,

growth failure and a profound lack of energy.

The STOPSC (Studies of Primary Sclerosing Cholangitis) registry

includes clinical information on children and adults with PSC,

autoimmune hepatitis and PSC/autoimmune hepatitis overlap syndrome.

Especially in children, there appears to be a relationship between

PSC and autoimmune hepatitis. The registry, which is based at Le

Bonheur Children's Medical Center, will comprise a database of

patient information and DNA samples for genetic studies. The STOPSC

project will facilitate collaborative, hypothesis-driven, multi-

center research on this rare disease. Le Bonheur is one of 13 major

North American medical centers encompassing 18 pediatric and adult

hepatology programs participating in the registry. The EMMES

Corporation in Rockville, MD, has been contracted to serve as the

STOPSC Data Coordinating Center.

Partners in this registry include:

University of Colorado, Denver

University of California, San Francisco

University of Cincinnati

Northwestern University, Chicago

Hospital for Sick Children, Toronto

Harvard, Boston

Mayo Clinic, Rochester

Mt. Sinai, New York

Le Bonheur Children's Medical Center and University of Tennessee

Health Science Center

Virginia Commonwealth, Richmond

Tufts, Boston

University of California,

University of Pittsburgh, Children's Hospital of Pittsburgh

Dr. Dennis Black is Co-Director of the Foundation and Study

Chair for STOPSC, as well as Le Bonheur Vice President for Research

and Director of the Children's Foundation Research Center at Le

Bonheur. " I'm excited that will help kick off the STOPSC

registry. This represents a major milestone in research for this

devastating disease. We hope that Le Bonheur sets the tone for this

collaborative relationship with our other partners across the US and

Canada, " Black said.

In addition to the major support from the Foundation, the

funding to begin the DNA repository portion of STOPSC was donated by

PSC Partners Seeking a Cure (www.pscpartners.org). PSC Partners

Seeking a Cure is a national PSC patient support organization founded

in 2005 to raise funds to research the causes and cure for PSC,

promote PSC and organ donation awareness and provide education and

support to PSC patients and their families.

The Foundation, founded in 2004 by Musette and ,

Jr., sponsors and facilitates basic and clinical research to discover

new treatments and ultimately a cure for primary sclerosing

cholangitis. Dr. Gene Whitington, a Le Bonheur pediatric

gastroenterologist who cares for the s' son, Worth, who has

PSC, was the driving force for the creation of the Foundation

and serves as its Director.

Posted: July 16, 2007

For more information please contact: Parris,

__________________

Dave

(father of (22); PSC 07/03; UC 08/03)