13th International Congress of Inborn Errors of Metabolism

Metabolic Diseases in the Plain People: the Path from Genomic Discovery to Therapy

Sept. 5 to 8, 2017
Rio de Janeiro, Brazil

On Tuesday, Sept. 5, this pre-congress workshop featured experts from Children's Hospital of Pittsburgh of UPMC and the Clinic for Special Children.

Chair: Jerry Vockley, MD, PhD
Co-chair: Lina Ghaloul-Gonzalez, MD

9 – 9:10 a.m. Introduction (Dr. Vockley, MD, PhD)
9:10 – 9:50 a.m. Genetic discovery among the Plain populations x precision medicine (Kevin Strauss, MD, Clinic for Special Children)
9:50 – 10:30 a.m. Determining the Best Liver Transplant Options in IEMs (Rakesh Sindhi, MD)
10:30 – 11 a.m. Coffee Break
11 – 11:40 a.m. Reduced Intensity Cord Blood or BMT for IEMs (Paul Szabolcs, MD)
11:40 a.m. – 12:20 p.m. Utilizing Natural History to Assess Outcomes for Novel Therapies (Maria Escolar, MD)
12:20 – 12:30 p.m. Wrap-Up (Lina Ghaloul-Gonzalez, MD)

For more information, please contact Jodie Vento, MGC, LCGC by phone at +1-412-692-RARE or 412-692-7273 or by email at RareCare@chp.edu.