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Course Outline

October 17, 2010-Day 0


– Preliminary Session


Course introduction (1 hr)
Overview of cellular intermediary metabolism, including chemical structures

Pre-course self-evaluation (1 hr)

October 18, 2010-Day 1


– Defects of Protein Metabolism


08:00   Laboratory Session I (amino acids, GC/MS, tandem MS) (1 hr)

09:00   Didactic Session I: Urea cycle disorders (45 min)

09:45   Didactic Integration Workshop I: Urea cycle disorders (45 min)

10:30   Break

11:00   Didactic Session II: Organic acidemias (1 hr)

12:00   Didactic Session III: Disorders of vitamin metabolism
           (cobalamin,folate, biotin, pyridoxine) (1 hr)

13:00   Lunch

14:00   Didactic Integration Workshop II: Organic acidemias (1 hr)

15:00   Didactic Session IV: Amino acid disorders (PKU, homocystinuria,
           tyrosinemia)(1 hr)

16:00   Break

16:30   Management Workshop I: Therapy for common protein metabolism
           disorders (1.5 hr)

18:30   Dinner

October 19, 2010-Day 2


– Energy Substrates and Hypoglycemia: Defects of Carbohydrate,
  Fatty Acid, and Ketone Body Metabolism


08:00   Didactic Session I: Introduction to hypoglycemia and gluconeogenesis
           (45 min)

08:45   Didactic Session II: Glycogen storage diseases (1 hr)

09:45   Break

10:00   Didactic Session III: Inborn errors of fructose and galactose metabolism
           (1 hr)

11:00   Didactic Session IV: Fatty acid oxidation defects and ketone body
           metabolism (1 hr)

12:00   Lunch

13:00   Didactic Integration Workshop I: Disorders of ketotic hypoglycemia
           (1.5 hr)

14:30   Didactic Integration Workshop II: Disorders of hypoketotic
           hypoglycemia (1.5 hr)

16:00   Break

16:30   Management Workshop I: Disorders of hypoglycemia and fasting
           intolerance (1.5 hr)

18:30   Dinner

October 20, 2010-Day 3:


- Mitochondrial Energy: Disorders of Oxidative Phosphorylation
  and Pyruvate Metabolism


- Defects of Metal Cofactors, Glycosylation, Creatine,
  Neurotransmitter Metabolism


08:00   Didactic Session I: Mitochondrial energy metabolism (45 min)

08:45   Didactic Session II: Diagnostic approach to lactic acidemia and defects
           of pyruvate metabolism (1 hr)

09:45   Break

10:15   Didactic Session III: Respiratory chain defects (1.15 hr)

11:30   Diagnosis and Management Workshop I: Mitochondrial energy
           metabolism disorders (1.5 hr)

13:00   Lunch

14:00   Didactic Session IV: Disorders of metal cofactors (copper, molybdenum)
           (1 hr)

15:00   Didactic Session V: Congenital disorders of glycosylation (1 hr)

16:00   Didactic Session VI: Disorders of creatine metabolism (30 min)

16:30   Break

17:00   Didactic Session VII: Defects of neurotransmitter metabolism (1 hr)

18:30   Dinner

October 21, 2010-Day 4


– Lysosomal Storage and Newborn Screening


08:00   Didactic Session I: Lysosomal storage diseases (30 min)

08:30   Didactic Session II: Radiology of inborn errors (1 hr)

09:30   Didactic Integration Workshop I: Physical manifestation of LSDs
           (somatic manifestations) (1 hr)

10:30   Break

11:00   Didactic Integration Workshop II: Neurological manifestions of LSDs
           (1 hr)

12:00   Lunch

13:00   Management Workshop I: Therapy of LSDs (1.5 hr)

14:30   Didactic Session III: Newborn Screening (1 hr)

15:30   Break

16:00   Philosophy and Style of Diagnosis (1.5 hr)

18:30   Dinner

October 22, 2010-Day 5


– Diagnostic Approaches: Radiologic, Laboratory, Newborn
  Screening and Peroxisomal Diseases


08:00   Didactic Session I: Neuroradiology (1 hr)

09:00   Didactic Session II: Peroxisomal biogenesis disorders (1 hr)

10:00   Break

10:30   Didactic Session III: Peroxisomal single enzyme disorders (1 hr)

11:30   Management Workshop 1: Peroxisomal disease (1 hr)

12:30   Lunch

13:30   Didactic Session IV: Dysmorphic features in inborn errors of
            metabolism (1hr)

14:30   Didactic Integration Workshop II: Approaching metabolic disease from
           a laboratory perspective (1.5 hr)

16:00   Break

16:30   Didactic Integration Workshop III: Evaluation of abnormal newborn
           screening results (1.5 hr)

18:30   Dinner

20:00   Jeopardy

October 23, 2010-Day 6


– Concluding Session


08:00   Final comments (15 min)

08:15   Course evaluation (15 min)

08:30   Post-course self-evaluation (1 hr)

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