L. Lawson

发表

Diaphragm Pacing as a Rehabilitative Tool for Patients With Pompe Disease Who Are Ventilator-Dependent: Case Series

B. Byrne, R. Onders, D. Fuller, 2016, Physical Therapy.

Phase I / II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase ( rAAV 1-CMV-GAA ) Gene Vector in Patients with Pompe Disease

T. Conlon, C. Mah, M. Corti, 2014 .

Respiratory motor function in individuals with centronuclear myopathies

B. Byrne, M. Corti, Barbara K. Smith, 2016, Muscle and Nerve.

Erratum: Respiratory Motor Function in Individuals with Centronuclear Myopathies

Arnp, B. Byrne, M. Corti, 2016, Muscle & nerve.

Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity

B. Byrne, M. Corti, A. Martin, 2017, Experimental Neurology.

B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease.

B. Byrne, R. Lawrence, R. Herzog, 2013, The Journal of pediatrics.

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease.

T. Conlon, B. Byrne, I. Tuna, 2017, Human gene therapy. Clinical development.

Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes.

T. Conlon, B. Byrne, D. Fuller, 2013, Human gene therapy.

The respiratory neuromuscular system in Pompe disease

B. Byrne, D. Fuller, M. ElMallah, 2013, Respiratory Physiology & Neurobiology.

Pompe disease gene therapy.

T. Conlon, B. Byrne, D. Fuller, 2011, Human molecular genetics.

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

T. Conlon, N. Clément, M. Corti, 2014, Molecular therapy. Methods & clinical development.

Development of a novel observer reported outcome tool as the primary efficacy outcome measure for a rare disease randomized controlled trial.

J. Shuster, S. Nixon, P. Stacpoole, 2017, Mitochondrion.

Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunction

B. Byrne, E. Christou, S. Subramony, 2015, Muscle & nerve.

Personalized Dosing of Dichloroacetate Using GSTZ1 Clinical Genotyping Assay.

Mohamed H. Shahin, P. Starostik, T. Langaee, 2018, Genetic testing and molecular biomarkers.