Barbara Smith, PT, PhD
Research Assistant Professor
Departments of Physical Therapy and Pediatrics
University of Florida
Phone: (352) 294-4058
B. Byrne, PhD
Respiratory strength and functional outcomes in patients with respiratory muscle weakness or mechanical ventilation. Use of exercise and regenerative rehabilitation to facilitate motor recovery of ventilation.
- 1992 Grove City College, Grove City, PA Molecular Biology BS
- 1994 University of Pittsburgh, Pittsburgh, PA Physical Therapy MPT
- 1998 University of Florida, Gainesville, FL Physical Therapy MHS
- 2010 University of Florida, Gainesville, FL Rehabilitation Science PhD
Grant#R21HD090752 (Smith) 09/01/16 - 08/31/18 National Institute of Child Health and Human Development (NICHD) Diaphragm Pacing and Rehabilitation in Pompe Disease Goal: Investigate acute and chronic effects of diaphragm pacing on the control of breathing and ventilator dependence in patients with Pompe disease. Role: Principal Investigator
Grant#1U01HL121842-01A1 (Byrne) 09/16/15-08/31/18 National Heart, Lung, and Blood Institute (NHLBI) Phase II Study of AAV9-GAA Gene Transfer in Pompe Disease. Goal: Evaluate the efficacy of systemic AAV9-GAA gene therapy in children with Pompe disease and ventilatory insufficiency. Role: Co-Investigator
Grant#NCT02704273 08/01/16-07/31/18 Audentes Therapeutics A Clinical Assessment Study in X-Linked Myotubular Myopathy Subjects (INCEPTUS) Goal: Provide important insights on the natural history of X-linked myotubular myopathy subjects aged 3 and younger. Role: Site Principal Investigator
Grant#NCT02453512 11/01/15-12/31/17 Audentes Therapeutics Respiratory Strength and Function in Untreated X-linked Myotubular Myopathy Goal: Evaluate the severity and progression of respiratory muscle function in patients with X-Linked Myotubular Myopathy (XLMTM) aged 0-14 years. Role: Principal Investigator
Grant#2U54AR052646-11 (Sweeney) 08/01/15-07/31/17 National Institutes of Health Failed Regeneration in the Muscular Dystrophies: Inflammation, Fibrosis, and Fat Goal: This project is organized around the concept that limiting fibrosis in the muscular dystrophies will help extend the period of successful regeneration. Role: Co-Investigator
Grant#NCT02354664 07/01/2015 - 2/28/2017 BioMarin Pharmaceutical Foundation Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease Goal: This study compares traditional tests of respiratory strength and endurance to magnetic resonance imaging as markers of dynamic respiratory motor function, in adults with late-onset Pompe disease. Role: Principal Investigator
Grant#NCT02191917 05/01/15-12/31/16 BioMarin Pharmaceutical A Study of Respiratory Muscle Strength in Patients with Late-Onset Pompe Disease Goal: Test respiratory muscle strength initially and again after 24 weeks, in subjects treated or not treated with BMN 701. Role: Principal Investigator
Grant#NCT02354651 03/01/14-02/28/17 Acid Maltase Deficiency Association Response to Diaphragmatic Pacing in Subjects with Pompe Disease Goal: Evaluate the natural history and quality of life in patients with Pompe disease who receive diaphragmatic pacemakers to augment ventilation. Role: PI
Grant#P50 GM111152 (Martin) 09/01/14-08/31/16 National Institutes of Health Diaphragm and Leg Strength Rehabilitation Strength in Chronically Critically Ill Patients Goal: Characterize the effect of persistent inflammation and catabolism syndrome on respiratory and limb muscle function, and evaluate the effect of strength training on muscle function, inflammation and catabolism. Role: Co-Investigator
Grant#11265471 (Byrne) 07/01/13-06/30/16 Department of Defense Intrapleural Microdystrophin Gene Therapy for Clinical Translation Goal: Determine the scalability and potency of AAV-microdystrophin gene therapy using a novel manufacturing platform and identify the efficacy of intrapleural delivery on cardiac and ventilatory muscle function, in a golden retriever model of Duchenne muscular dystrophy. Role: Co-Investigator
Grant#CRB-HLBI1-S-10-00223 (Byrne) 10/01/10-10/30/15 National Institutes of Health Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease Goal: Evaluate the safety of the experimental gene transfer procedure in individuals with GAA deficiency, and determine what dose may be required to achieve improvement in measures of respiratory function. Role: Co-Investigator
Grant#K12 HD055929-05 (Ottenbacher) 09/25/07-08/31/17 National Center for Medical Rehabilitation Research (NICHD), NIH Rehabilitation Research Career Development Program Goal: Recruit and train physical and occupational therapy faculty scholars in rehabilitation research to prepare them for successful independent research careers. Role: Scholar (2011-13)
Grant#NCT02453512 (Smith) 11/01/15 - 12/31/17 Audentes Therapeutics, Inc. Respiratory Strength and Function in Untreated X-linked Myotubular Myopathy Goal: This study is a longitudinal study evaluating the severity and progression of respiratory muscle function in patients with X-Linked Myotubular Myopathy (XLMTM) aged 0-14 years.
Grant#NCT02354664 (Smith) 07/01/15 - 12/31/16 BioMarin Pharmaceutical Foundation Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease (NCT02354664) Goal: This study compares traditional tests of respiratory strength and endurance to magnetic resonance imaging as markers of dynamic respiratory motor function, in adults with late-onset Pompe disease.
Grant#NCT02191917 (Smith, Site PI) 05/01/15 - 12/31/16 BioMarin Pharmaceutical Goal: A Study of Respiratory Muscle Strength in Patients with Late-onset Pompe Disease Study 701-201 is a study in patients with late-onset Pompe disease (LOPD). The study will test respiratory muscle strength initially and again after 24 weeks, in subjects treated or not treated with BMN 701.
Grant#AMDA (Smith) 03/01/14 - 02/28/17 Diaphragm Pacing and Quality of Life in Patients with Pompe Disease. This project characterizes ventilatory strength and electrophysiological properties of phrenic motor units during voluntary and evoked diaphragm contractions, in adults with Pompe disease.
Grant# 2U54AR052646 (Sweeney) 08/01/15 - 07/31/17 NIH Failed Regeneration in the Muscular Dystrophies: Inflammation, Fibrosis, and Fat Goal: This project is organized around the concept that limiting fibrosis in the muscular dystrophies will help extend the period of successful regeneration.
Grant#U01HL121842 (Byrne) 10/01/15 - 12/31/18 NIH/NHLBI Phase II Study of AAV9-GAA Gene Transfer in Pompe Disease Goal: The study investigates advancement of clinical research of adeno-associated vector (AAV) expressing human acid alpha-glucosidase (GAA) approaches to treat respiratory failure caused by Pompe disease.
Grant# DOD- MD120087 (Byrne) 07/01/13 - 06/30/16 Intrpleural Microdystrophin Gene Therapy for Clinical Translation. Goal: This project will characterize safety and cardiopulmonary function following treatment with microdystrophin gene therapy, in a canine model of Duchenne muscular dystrophy. Role: Co-I, funded for years 2-3.
Publications (past 5 years)
Mankowski RT, Ahmed S, Beaver T, Marvin D, Chul H, Hess P, Martin T, Smith BK, Someya S, Leeuwenburgh A, Martin AD. Intraoperative hemidiaphragm electrical stimulation reduces oxidative stress and upregulates autophagy in surgery patients undergoing mechanical ventilation- exploratory study. Journal of Translational Medicine. In Press.
Smith BK, Martin AD, Lawson LA, Vernot V, Marcus J, Islam S, Shafi N, Corti M, Collins SW, Byrne BJ. Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: clinical evidence of respiratory plasticity. Exp Neurol. 2017 Jan;287(Pt 2):216-224. doi: 10.1016/j.expneurol.2016.07.013.
Smith BK, Fuller DD, Martin AD, Lottenberg L, Islam S, Lawson LA, Onders RP, Byrne BJ. Diaphragm pacing in ventilator dependent Pompe disease. Physical Therapy. 2016; 96(5), 696-703. PMCID: PMC4858660.
Smith BK, Corti M, Martin AD, Fuller DD, Byrne BJ. Altered activation of the diaphragm in late-onset Pompe disease. Respiratory Physiology & Neurobiology. 2016; 222, 11-5. PMCID: PMC4869700.
Smith BK, Renno M, Green MM, Sexton T, Martin D, Corti M, Byrne BJ. Respiratory motor function in individuals with centronuclear myopathy and ventilatory insufficiency. Muscle and Nerve. 2016; 52(2), 214-21. PMCID: PMC4718843.
Goddard MA, Mack DL, Hogrel JY, Czerniecki SM, Kelly VE, Snyder JM, Grange RW, Lawlor MW, Smith BK, Beggs AH, Childers MK. Muscle pathology, limb strength, walking gait, respiratory function and neurological impairment establish disease progression in the p.N155K canine model of X-linked myotubular myopathy. Ann Transl Med 2015; 3(18), 262. PMCID: PMC4630545.
Corti M, Smith BK, Falk D, Fuller DD, Subramony SH, Byrne BJ, Christou EA. Altered activation of the tibialis anterior in individuals with Pompe disease: implications for motor unit dysfunction. Muscle and Nerve. 2015; 51(6), 877-83. PMCID: PMC4348349.
Corti M, Elder M, Falk D, Lawson LA, Smith BK, Nayak B, Conlon T, Clement N, Erger K, Lavassani E, Green M, Doerfler P, Herzog R, Byrne BJ. B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study. Molecular Therapy Methods: Clinical Development. 2014; 1, pii: 14033. PMCID: PMC4275004.
Byrne BJ, Collins S, Mah C, Smith B, Conlon T, Martin AD, Corti M, Cleaver B, Islam S, Lawson LA. Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAV1-CMVGAA) gene vector in patients with Pompe disease. Human Gene Therapy: Clinical Development. 2014; 25(3), 143-63.
Smith BK, Mathur S, Ye F, Martin AD, Truelson S, Vandenborne K, Davenport PW. Intrinsic Transient Trachial Occlusion Training Facilitates Myogenic Remodeling in Rodent Parasternal Intercostal Fibers. Journal of Rehabilitation Research and Development. 2014; 51(5), 841-54. PMCID: PMC4269303.
Ahn B, Beaver T, Martin T, Hess P, Brumback BA, Ahmed S, Smith BK, Leeuwenburgh C, Martin AD, Ferreira LF. Phrenic nerve stimulation increases human diaphragm fiber force after cardiothoracic surgery. American Journal of Respiratory and Critical Care Medicine. 2014; 190(7), 837-9. PMCID: PMC4299610.
Ye F, McCoy SC, Ross HH, Bernardo JA, Beharry AW, Senf SA, Judge AR, Beck DT, Conover CF, Cannady DF, Smith BK, Yarrow JF, Borst SE. Transcriptional regulation of myotrophyic actions by testosterone and trenbolone on androgen-responsive muscle. Steroids. 2014; 87, 59-66.
Smith BK, Goddard M, Childers MK. Respiratory assessment in centronuclear myopathies. Muscle and Nerve. 2014; 50(3), 315-26. PMCID: PMC4140950.
Martin AD, Joseph AM, Beaver TM, Smith BK, Martin TD, Berg K, Hess PK, Deoghare HV, Leeuwenburgh, C. Effect of intermittent phrenic nerve stimulation during cardiothoracic surgery on mitochondrial respiration in the human diaphragm. Critical Care. 2014; 42(2), e152-6. PMCID: PMC3947040.
Smith BK, Gabrielli A, Davenport PW, Martin AD. Effect of Training on Inspiratory Load Compensation in Weaned and Unweaned Mechanically Ventilated ICU Patients. Respiratory Care. 2014: 59(1), 22-31. PMCID: PMC4157996.
Mah CS, Soustek MS, Todd AG, McCall A, Smith BK, Corti M, Falk DJ, Byrne BJ. Adeno-Associated Virus- Mediated Gene Therapy for Metabolic Myopathy. Human Gene Therapy. 2013; 24(11), 928-36. PMCID: PMC3814817.
Fuller DD, Elmallah M, Smith BK, Corti M, Lawson LA, Falk DJ, Byrne BJ. The Respiratory System in Pompe Disease. Resp Physiol Neurobiol. 2013; 189(2), 241-9. PMCID: PMC4083814.
Martin AD, Smith BK, Gabrielli, A. Mechanical ventilation, diaphragm weakness and weaning: a rehabilitation perspective. Resp Physiol Neurobiol. 2013; 189(2), 377-83. PMCID: PMC3808482.
Smith BK, Mah CS, Martin AD, Lawson LA, Conlon TJ, Clement N, Islam S, Dobjia N, Collins SW, Byrne BJ. Phase I/II trial of AAV1-GAA gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes. Human Gene Therapy, 2013; 24(6), 630-40. PMCID: PMC3689178.
Smith BK, Bleiweis MS, Neel C, Martin AD. Inspiratory Muscle Strength Training in Infants with Congenital Heart Disease and Prolonged Mechanical Ventilation: Case Report. Physical Therapy. 2013; 93(2), 229-36. PMCID: PMC3563028.
Smith BK, Martin AD, Vandenborne K, Darragh BD, Davenport PW. Chronic intrinsic transient tracheal occlusion elicits diaphragm remodeling in conscious rodents. PLoS One. 2012; 7(11), e29624. PMCID: PMC3486807.
Goddard MA, Mitchell EL, Smith BK, Childers MK. Establishing Clinical End Points of Respiratory Function in Large Animals for Clinical Translation. Physical Medicine and Rehabilitation Clinics of North America, 2012; 23(1), 75-94.
Huang T, Deoghare HV, Smith BK, Beaver TM, Baker HV, Martin AD. Gene Expression Changes in the Human Diaphragm Following Cardiothoracic Surgery. Journal of Thoracic and Cardiovascular Surgery. 2011; 142(5), 1214-22.
Byrne BJ, Falk DJ, Pacak CA, Nayak S, Hersog RW, Elder ME, Collins SW, Conlon TJ, Clement N, Cleaver BD, Cloutier DA, Porvasnik SL, Islam S, Elmallah MK, Martin A, Smith BK, Fuller DD, Lawson LA, Mah CS. Pompe disease gene therapy. Human Molecular Genetics. 2011; 20(R1), R61-8. PMCID: PMC3095055.
Edwards TM, Smith BK, Watts DL, Germain-Aubrey CC, Roark A, Bybee S, Cox CE, Hamlin HJ, Guillette LJ. Group-Advantaged Training of Research (G.A.T.O.R.): A Metamorphosis of Mentorship. Bioscience. 2011; 61(4), 301-311.
Martin AD, Smith BK, Davenport PW, Harman E, Gonzalez-Rothi R, Baz M, Layon J, Banner M, Caruso L, Deoghare H, Huang T, Gabrielli A. Inspiratory muscle strength training improves weaning outcome in failure to wean patients: A controlled trial. Critical Care. 2011; 15(2), R84. PMCID: PMC3219341.
Smith BK, Bleiweis MS, Zauhar J, Martin AD. Inspiratory Muscle Training in a Child with Nemaline Myopathy and Organ Transplantation. Pediatric Critical Care Medicine, 2011; 12(2), e94-8. PMCID: PMC3633211.
Complete List of Published Work in MyBibliography