Carolyn T. Spencer

Pompe disease diagnosis and management guideline

Priya S. Kishnani, Robert D. Steiner, Deeksha Bali et al. · Genetics in Medicine · 2006 · 628 citations · Full text

Veterinary Medicine, Pathobiology, Postharvest Disease Management +2

Cardiac and Clinical Phenotype in Barth Syndrome

Carolyn T. Spencer, Randall M. Bryant, Jane Day et al. · PEDIATRICS · 2006 · 235 citations

Medicine, Cardiology, Molecular Genetics +13

Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2 <i>α</i> 1-Antitrypsin (AAT) Vector in AAT-Deficient Adults

Mark Brantly, L. Terry Spencer, Margaret Humphries et al. · Human Gene Therapy · 2006 · 175 citations

Pharmacy, Pharmaceutical Biotechnology, Pharmacotherapy +14

Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV2-CB-hAAT) Gene Vector to AAT-Deficient Adults

Terence R. Flotte, Mark Brantly, L. Terry Spencer et al. · Human Gene Therapy · 2004 · 146 citations

Pharmacy, Molecular Biology, Pharmaceutical Biotechnology +14

The Barth Syndrome Registry: Distinguishing disease characteristics and growth data from a longitudinal study

Amy E. Roberts, Connie S. Nixon, Colin G. Steward et al. · American Journal of Medical Genetics Part A · 2012 · 142 citations

Life Sciences, Genetics, Pediatrics +16