CONCURRENT SESSION III -  MALABSORPTION - PROTEIN LOSING ENTEROPATHIES

MUTATIONS IN DGAT1 CAUSE PROTEIN LOSING ENTEROPATHY
Harland Winter MD, Mass General Hospital
Learning objectives:
At the end of the presentation, participants should be able to:
1. Recognize when to evaluate a patient for a mutation in DGAT1
2. Diagnose patients who have DGAT1 mutations
3. Prescribe appropriate treatment for patients with DGAT1 mutations
Harland Winter MD - Slides
(Adobe PDF File)
EFFICACY, GROWTH, AND SAFETY OUTCOMES OF TEDUGLUTIDE IN CHILDREN WITH SHORT BOWEL SYNDROME–ASSOCIATED INTESTINAL FAILURE (SBS–IF): A PHASE 3 STUDY. Samuel Kocoshis1, Beth Carter2,
Susan Hill3, Simon Horslen4, Stuart Kaufman5, David Mercer6, Russell Merritt7, Mikko Pakarinen8, Susan Protheroe9, Robert Venick10, Paul W. Wales11, Simin Hu12, Andrew Grimm12. 1Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA; 2Children’s Hospital of Los Angeles, Keck USC School of Medicine, Los Angeles, CA, USA; 3Great Ormond Street Hospital for Children, London, United Kingdom; 4Seattle Children’s Hospital and University of Washington School of Medicine, Seattle, WA, USA; 5MedStar Georgetown Transplant Institute and Georgetown University School of Medicine, Washington, , USA; 6Nebraska Medical Center, Omaha, NE, USA; 7Children’s Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, CA, USA; 8Children’s Hospital, Helsinki University Central Hospital, Helsinki, Finland; 9Birmingham Women’s and Children’s Hospital NHS Trust, Birmingham, United Kingdom; 10Mattel Children’s Hospital UCLA, Los Angeles, CA, USA; 11The Hospital for Sick Children, Toronto, ON, Canada; 12Shire Human Genetic Therapies, Inc., Lexington, MA, USA.
Samuel Kocoshis MD - Slides
(Adobe PDF File)
MYSTERIES OF PROTEIN LOSING ENTEROPATHY UNRAVELED
Timothy Sentongo MD, University of Chicago
Learning objectives:
1. Recognize the presentation and differential diagnosis of non-genetic causes of protein losingenteropathy
2. Understand the mechanism and pathophysiology of protein losing enteropathies
3. Learn the management of challenging cases of protein losing enteropathy
Timothy Sentongo MD - Slides
(Adobe PDF File)
AN ENTEROID MODEL FO CONGENITAL TUFTING ENTEROPATHY SHOWS ALTERATION IN INTESTINAL
EPITHELIAL CELL DIFFERENTIATION. Barun Das1, Kevin Okamoto1, John Rabalais1, Soumita Das2, Mamata Sivagnanam1. 1Pediatrics, UCSD/Rady Children's Hospital, La Jolla, CA, USA; 2Pathology, UCSD, La Jolla, CA, USA.
Mamata Sivagnanam - Slides
(Adobe PDF File)