Search Results - "Spencer, Carolyn"

The Barth Syndrome Registry: Distinguishing disease characteristics and growth data from a longitudinal study by Roberts, Amy E., Nixon, Connie, Steward, Colin G., Gauvreau, Kimberly, Maisenbacher, Melissa, Fletcher, Matthew, Geva, Judith, Byrne, Barry J., Spencer, Carolyn T.

“…Barth syndrome (BTHS); MIM accession # 302060) is a rare X‐linked recessive cardioskeletal mitochondrial myopathy with features of cardiomyopathy, neutropenia,…”
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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy by Brantly, Mark L, Chulay, Jeffrey D, Wang, Lili, Mueller, Christian, Humphries, Margaret, Spencer, L. Terry, Rouhani, Farshid, Conlon, Thomas J, Calcedo, Roberto, Betts, Michael R, Spencer, Carolyn, Byrne, Barry J, Wilson, James M, Flotte, Terence R

“…Alpha-1 antitrypsin (AAT) deficiency is well-suited as a target for human gene transfer. We performed a phase 1, open-label, dose-escalation clinical trial of…”
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Cardiac and Clinical Phenotype in Barth Syndrome by Spencer, Carolyn T, Bryant, Randall M, Day, Jane, Gonzalez, Iris L, Colan, Steven D, Thompson, W. Reid, Berthy, Julie, Redfearn, Sharon P, Byrne, Barry J

“…Barth syndrome, an X-linked disorder that is characterized by cardiomyopathy, neutropenia, skeletal myopathy, and growth delay, is caused by mutations in the…”
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Reproducibility of Echocardiographic Diagnosis of Left Ventricular Noncompaction by Saleeb, Susan F., MD, Margossian, Renee, MD, Spencer, Carolyn T., MD, Alexander, Mark E., MD, Smoot, Leslie B., MD, Dorfman, Adam L., MD, Bergersen, Lisa, MD, MPH, Gauvreau, Kimberlee, ScD, Marx, Gerald R., MD, Colan, Steven D., MD

“…Background Left ventricular noncompaction (LVNC) cardiomyopathy is variably defined by numerous trabeculations, deep intertrabecular recesses, and…”
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Pompe disease diagnosis and management guideline by Kishnani, Priya S, Steiner, Robert D, Bali, Deeksha, Berger, Kenneth, Byrne, Barry J, Case, Laura E, Case, Laura, Crowley, John F, Downs, Steven, Howell, R Rodney, Kravitz, Richard M, Mackey, Joanne, Marsden, Deborah, Martins, Anna Maria, Millington, David S, Nicolino, Marc, O'Grady, Gwen, Patterson, Marc C, Rapoport, David M, Slonim, Alfred, Spencer, Carolyn T, Tifft, Cynthia J, Watson, Michael S

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Substrate metabolism during basal and hyperinsulinemic conditions in adolescents and young-adults with Barth syndrome by Cade, W. Todd, Spencer, Carolyn T., Reeds, Dominic N., Waggoner, Alan D., O’Connor, Robert, Maisenbacher, Melissa, Crowley, Jan R., Byrne, Barry J., Peterson, Linda R.

“…Background Barth syndrome (BTHS) is a rare X-linked disorder that is characterized by mitochondrial abnormalities, infantile or childhood onset of…”
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Barth syndrome by Clarke, Sarah L N, Bowron, Ann, Gonzalez, Iris L, Groves, Sarah J, Newbury-Ecob, Ruth, Clayton, Nicol, Martin, Robin P, Tsai-Goodman, Beverly, Garratt, Vanessa, Ashworth, Michael, Bowen, Valerie M, McCurdy, Katherine R, Damin, Michaela K, Spencer, Carolyn T, Toth, Matthew J, Kelley, Richard I, Steward, Colin G

“…First described in 1983, Barth syndrome (BTHS) is widely regarded as a rare X-linked genetic disease characterised by cardiomyopathy (CM), skeletal myopathy,…”
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Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults by Flotte, Terence R, Brantly, Mark L, Spencer, L Terry, Byrne, Barry J, Spencer, Carolyn T, Baker, Dawn J, Humphries, Margaret

“…A recombinant virus vector constructed from adeno-associated virus (AAV) that has been altered to carry the human alpha1-antitrypsin (hAAT) gene expressed from…”
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Estimating Equations for Cardiopulmonary Exercise Testing Variables in Fontan Patients: Derivation and Validation Using a Multicenter Cross-Sectional Database by Butts, Ryan J., Spencer, Carolyn T., Jackson, Lanier, Heal, Martha E., Forbus, Geoffrey, Hulsey, Thomas C., Atz, Andrew M.

“…Cardiopulmonary exercise testing (CPET) is a common method of evaluating patients with a Fontan circulation. Equations to calculate predicted CPET values are…”
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Impact of Initial Shunt Type on Cardiac Size and Function in Children With Single Right Ventricle Anomalies Before the Fontan Procedure by Frommelt, Peter C., MD, Gerstenberger, Eric, MS, Cnota, James F., MD, Cohen, Meryl S., MD, Gorentz, Jessica, RDCS, Hill, Kevin D., MD, MS, John, J. Blaine, MD, Levine, Jami C., MD, Lu, Jimmy, MD, Mahle, William T., MD, McCandless, Rachel T., MD, Mertens, Luc, MD, PhD, Pearson, Gail D., MD, ScD, Spencer, Carolyn, MD, Thacker, Deepika, MD, Williams, Ismee A., MD, MS, Wong, Pierre C., MD, Newburger, Jane W., MD, MPH

“…Abstract Background In children with single right ventricular (RV) anomalies, changes in RV size and function may be influenced by shunt type chosen at the…”
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REPRODUCIBILITY OF ECHOCARDIOGRAPHIC DIAGNOSIS OF VENTRICULAR NONCOMPACTION by Margossian, Renee, Spencer, Carolyn T, Alexander, Mark E, Smoot, Leslie B, Dorfman, Adam L, Bergersen, Lisa, Gauvreau, Kimberlee, Marx, Gerald R, Colan, Steven D

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1060 Evaluation of aortic valve regurgitation by cardiac magnetic resonance imaging: a comparison with echocardiography by Surenderanath, Nisha, Al-Mousily, Faris, Theriaque, Douglas, Shuster, Jonathan, Spencer, Carolyn, Samyn, Margaret M

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Impaired cardiac reserve and severely diminished skeletal muscle O₂ utilization mediate exercise intolerance in Barth syndrome by Spencer, Carolyn T, Byrne, Barry J, Bryant, Randall M, Margossian, Renee, Maisenbacher, Melissa, Breitenger, Petar, Benni, Paul B, Redfearn, Sharon, Marcus, Edward, Cade, W Todd

“…Barth syndrome (BTHS) is a mitochondrial myopathy characterized by reports of exercise intolerance. We sought to determine if 1) BTHS leads to abnormalities of…”
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728. Safety and Bioactivity of rAAV2-hAAT in alpha-1 Antitrypsin-Deficient Patients in a Phase I Clinical Trial by Brantly, Mark L., Spencer, Terry, Spencer, Carolyn T., Conlon, Thomas J., Song, Sihong, Humphries, Margaret, Byrne, Barry J., Snyder, Richard O., Flotte, Terence R.

“…INTRODUCTION: Recombinant adeno-associated virus (rAAV) vectors are capable of sustained expression of therapeutic proteins when administered to skeletal…”
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Total parenteral nutrition, an ally in the management of patients with intestinal failure and malnutrition: a long-term view by Spencer, CT, Compher, CW

“…Dr. Jonathan Rhoads played a key role in the development of parenteral nutrition, a feeding method used today for the same clinical conditions as during…”
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Intersecting Identities, Education, and Economic and Subjective Well-Being: A Qualitative "Testimonio" Study among Latina Single Mother Community College Students by Peguero Spencer, Carolyn

“…The COVID-19 pandemic has laid bare the gender inequities of unpaid household and childcare labor, especially for Black and Latina mothers who are also…”
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An institutional review of the value of computed tomographic angiography in the diagnosis of congenital cardiac malformations by Chandran, Arun, Fricker, F Jay, Schowengerdt, Kenneth O, Cumming, William A, Saidi, Arwa, Spencer, Carolyn T, Paolillo, Joseph, Samyn, Margaret M

“…The ultra-fast, thin-cut computerised tomographic angiogram is an efficient method to diagnose extracardiac lesions associated with congenital cardiac disease…”
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Investigating Diversity in Social Work Doctoral Education in the United States by Chin, Matthew, Hawkins, Jaclynn, Krings, Amy, Peguero-Spencer, Carolyn, Gutiérrez, Lorraine

“…Despite its emphasis on social justice, social work in the United States has not always attended to issues of diversity in doctoral education. This article…”
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Impact of Initial Shunt Type on Cardiac Size and Function in Children With Single Right Ventricle Anomalies Before the Fontan Procedure: The Single Ventricle Reconstruction Extension Trial by FROMMELT, Peter C, GERSTENBERGER, Eric, MCCANDLESS, Rachel T, MERTENS, Luc, PEARSON, Gail D, SPENCER, Carolyn, THACKER, Deepika, WILLIAMS, Ismee A, WONG, Pierre C, NEWBURGER, Jane W, CNOTA, James F, COHEN, Meryl S, GORENTZ, Jessica, HILL, Kevin D, JOHN, J. Blaine, LEVINE, Jami C, LU, Jimmy, MAHLE, William T

“…In children with single right ventricular (RV) anomalies, changes in RV size and function may be influenced by shunt type chosen at the time of the Norwood…”
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Echocardiographic findings in patients diagnosed with glycoproteinoses diseases by Pietris, Nicholas, Cathey, Sara, Spencer, Carolyn

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