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Jerry Vockley

Division Director, Genetic and Genomic Medicine, Cleveland Family Endowed Chair in Pediatric Research · Univ of Pittsburgh Sch of Med

Credentials: · · · · ·

History

and Director · Univ of Pittsburgh Sch of Med
Present
Division of Genetic and Genomic Medicine · UPMC Medical Education
Present
Center for Rare Disease Therapy · UPMC Medical Education
Present

Pediatrics
Yale Univ Sch of Med
1987–1991
Pediatrics
University of Colorado School of Medicine
1984–1987
University of Pennsylvania School of Medicine
The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania
1980–1984
Biology, Carnegie Mellon University
1974–1978

Research

Increased levels of plasma acylcarnitines in obesity and type 2 diabetes and identification of a marker of glucolipotoxicity
Obesity · 2010 · 552 citations
Phenylalanine hydroxylase deficiency: diagnosis and management guideline
Genetics in Medicine · 2014 · First of 11 authors · 510 citations
Effects of diet and physical activity interventions on weight loss and cardiometabolic risk factors in severely obese adults: a randomized trial
JAMA · 2010 · 406 citations

Barbara Burton
26 papers
Nicola Longo
26 papers
CH
Cary Harding
24 papers
Lina Gonzalez
18 papers
George Mazariegos
16 papers
Amy Goldstein
15 papers
RZ
Roberto Zori
14 papers
Stephen Cederbaum
14 papers
Susan Berry
14 papers
PR
Piero Rinaldo
13 papers · 2 as a resident
Uta Lichter
13 papers
Wendy Smith
12 papers

Grants

Branched Chain Amino Acid Metabolism and Citrate Homeostasis in Disease
National Institute of Diabetes and Digestive and Kidney Diseases · R01
Contact PI · $764.2K · University of Pittsburgh at Pittsburgh
2026–2031
Rare Organic Acidemias Research (ROAR) Consortium
National Institute of Child Health and Human Development · U54
Project lead · $298.1K · Baylor College of Medicine
2025–2030
Therapeutic Genetics and Disease Modeling in LAMA2-CMD
National Institute of Arthritis and Musculoskeletal and Skin Diseases · R01
Contact PI · $1.3M · University of Pittsburgh at Pittsburgh
2022–2027
Characterization of Branched Chain Amino Acid Metabolism and Its Deficiency
National Institute of Diabetes and Digestive and Kidney Diseases · R01
Contact PI · $4.1M · University of Pittsburgh at Pittsburgh
2016–2026

Clinical trials

Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Principal investigator · Withdrawn · Phase 2
Jerry Vockley, MD, PhD
2026–2029
Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Principal investigator · Withdrawn · Phase 2
Jerry Vockley, MD, PhD
2026–2028
An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)
Principal investigator · Enrolling by invitation · Phase 1
Jerry Vockley, MD, PhD
2025–2027
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Principal investigator · Recruiting · Phase 2
Jerry Vockley, MD, PhD
2025–2027