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Bardet Biedl Syndrome Foundation

Connecting families and sharing information on research, treatment, and therapies for Bardet Biedl Syndrome.

From the BBS Center of Excellence

A letter to BBS families from Dr. Bob Haws, director of the BBS Center of Excellence at the Marshfield Clinic

Dear BBS family members,

As a physician caring for children with BBS I am impressed that a leading priority for you and others is preserving vision for your loved one or yourself. As a father of a wonderful daughter affected by blindness I understand your passion. A second priority for individuals with BBS is prevention of obesity by appetite control. The two issues of loss of vision and appetite control are critical to almost every child with BBS. The University of Iowa has published encouraging research reporting that tauroursodeoxycholic acid (TUDCA) slowed retinal degeneration in an animal model of BBS. Furthermore TUDCA resulted in decreased food consumption and weight loss in the animals.

A clinical trial of TUDCA in individuals with BBS has been proposed but not yet initiated. Such clinical trials are very expensive. Dr. Arlene Drack at the University of Iowa is pursuing funding. The Foundation Fighting Blindness has expressed interest in considering TUDCA research. I strongly encourage you to write Dr. Steve Rose, Chief Science Officer at the Foundation Fighting Blindness expressing support, and your family’s interest in participating in a clinical trial of TUDCA in individuals with BBS. Contact information for the Foundation Fighting Blindness is provided below:

Foundation Fighting Blindness
7168 Columbia Gateway Drive, Suite 100
Columbia, MD 21046
Toll Free: (800) 683-5555

Important points to consider in your communication may include the following:

1) The treatment results with TUDCA in an appropriate animal model of BBS are extremely encouraging. Translational research, taking research from laboratory animals to humans, is critical. This research is integral
to the objective of the FFB.

2) Bardet-Biedl syndrome provides an important disease model for other degenerative retinal diseases. Successful identification of effective therapy in BBS may be applicable to other diseases causing blindness.

3) The Bardet-Biedl Syndrome community is a growing community with a commitment to research. You may consider stating that you represent one individual anxious to support and participate in this research.

4) TUDCA provides hope to the BBS community not only for the potential benefit on vision but also for the appetite suppressing effect that will improve the quality of life for individuals with BBS.

Please take time to send your support for this research. Your voices are extremely powerful and need to be heard.

While we are hopeful for a TUDCA clinical trial, remember that there are no reliable and safe sources of TUDCA available for individuals today. Attempting to self-medicate with TUDCA is dangerous.

Bob Haws, M.D.
Pediatric Nephrology
Medical Director
Treatment Center for Bardet-BIedl Syndrome and Related Ciliopathies