Search

Your search keyword '"Tennekoon, G."' showing total 25 results
Start Over Author "Tennekoon, G." Search Limiters Peer Reviewed
25 results on '"Tennekoon, G."'

1. Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy

Author

Bernert, G., Knipp, F., Buyse, G.M., Goemans, N., Van den Hauwe, M., Voit, T., Doppler, V., Gidaro, T., Cuisset, J.-M., Coopman, S., Schara, U., Lutz, S., Kirschner, J., Borell, S., Will, M., D'Angelo, M.G., Brighina, E., Gandossini, S., Gorni, K., Falcier, E., Politano, L., D'Ambrosio, P., Taglia, A., Verschuuren, J.J.G.M., Straathof, C.S.M., Vílchez Padilla, J.J., Muelas Gómez, N., Sejersen, T., Hovmöller, M., Jeannet, P.-Y., Bloetzer, C., Iannaccone, S., Castro, D., Tennekoon, G., Finkel, R., Bönnemann, C., McDonald, C., Henricson, E., Joyce, N., Apkon, S., Richardson, R.C., Meier, Thomas, Rummey, Christian, Leinonen, Mika, Spagnolo, Paolo, Mayer, Oscar H., and Buyse, Gunnar M.

Published
2017
Full Text
View/download PDF

2. Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy

Author

Bernert, G., Knipp, F., Buyse, G.M., Goemans, N., van den Hauwe, M., Voit, T., Doppler, V., Gidaro, T., Cuisset, J.-M., Coopman, S., Schara, U., Lutz, S., Kirschner, J., Borell, S., Will, M., D'Angelo, M.G., Brighina, E., Gandossini, S., Gorni, K., Falcier, E., Politano, L., D'Ambrosio, P., Taglia, A., Verschuuren, J.J.G.M., Straathof, C.S.M., Vílchez Padilla, J.J., Muelas Gómez, N., Sejersen, T., Hovmöller, M., Jeannet, P.-Y., Bloetzer, C., Iannaccone, S., Castro, D., Tennekoon, G., Finkel, R., Bönnemann, C., McDonald, C., Henricson, E., Joyce, N., Apkon, S., Richardson, R.C., McDonald, Craig M., Meier, Thomas, Voit, Thomas, Schara, Ulrike, Straathof, Chiara S.M., D'Angelo, M. Grazia, Bernert, Günther, Cuisset, Jean-Marie, Finkel, Richard S., Goemans, Nathalie, Rummey, Christian, Leinonen, Mika, Spagnolo, Paolo, and Buyse, Gunnar M.

Published
2016
Full Text
View/download PDF

5. Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

Author

Conrado, D. J., Larkindale, J., Berg, A., Hill, M., Burton, J., Abrams, K. R., Abresch, R. T., Bronson, A., Chapman, D., Crowther, M., Duong, T., Gordish-Dressman, H., Harnisch, L., Henricson, E., Kim, S., Mcdonald, C. M., Schmidt, S., Vong, C., Wang, X., Wong, B. L., Yong, F., Romero, K., Vishwanathan, V., Chidambaranathan, S., Douglas Biggar, W., Mcadam, L. C., Mah, J. K., Tulinius, M., Cnaan, A., Morgenroth, L. P., Leshner, R., Tesi-Rocha, C., Thangarajh, M., Kornberg, A., Ryan, M., Nevo, Y., Dubrovsky, A., Clemens, P. R., Abdel-Hamid, H., Connolly, A. M., Pestronk, A., Teasley, J., Bertorini, T. E., Webster, R., Kolski, H., Kuntz, N., Driscoll, S., Bodensteiner, J. B., Gorni, K., Lotze, T., Day, J. W., Karachunski, P., Henricson, E. K., Joyce, N. C., Campbell, C., Torricelli, R. E., Finkel, R. S., Flanigan, K. M., Goemans, N., Heydemann, P., Kaminska, A., Kirschner, J., Muntoni, F., Osorio, A. N., Schara, U., Sejersen, T., Shieh, P. B., Sweeney, H. L., Topaloglu, H., Vilchez, J. J., Voit, T., Wong, B., Alfano, L. N., Eagle, M., James, M. K., Lowes, L., Mayhew, A., Mazzone, E. S., Nelson, L., Rose, K. J., Abdel-Hamid, H. Z., Apkon, S. D., Barohn, R. J., Bertini, E., Bloetzer, C., Devaud, L. C., Butterfield, R. J., Chabrol, B., Chae, J. H., Jongno-Gu, D. R., Comi, G. P., Darras, B. T., Dastgir, J., Desguerre, I., Escobar, R. G., Finanger, E., Guglieri, M., Hughes, I., Iannaccone, S. T., Jones, K. J., Kudr, M., Mathews, K., Parsons, J., Pereon, Y., de Queiroz Campos Araujo, A. P., Renfroe, J. B., de Resende, M. B. D., Selby, K., Tennekoon, G., Vita, G., Apkon, S., Barohn, R., Belousova, E., Brandsema, J., Bruno, C., Burnette, W., Butterfield, R., Byrne, B., Carlo, J., Chandratre, S., Comi, G., Connolly, A., De Groot I, I., Deconinck, N., Dooley, J., Durigneux, J., Finkel, R., Frank, L. M., Harper, A., Hattori, A., Herguner, O., Iannaccone, S., Janas, J., Jong, Y. J., Komaki, H., Lee, W. T., Leung, E., Mah, J., Mercuri, E., Mcmillan, H., Mueller-Felber, W., de Munain A, L., Nakamura, A., Niks, E., Ogata, K., Pascual, S., Pegoraro, E., Renfroe, B., Sanka, R. B., Schallner, J., Sendra, I. I., Servais, L., Smith, E., Sparks, S., Victor, R., Wicklund, M., Wilichoswki, E., Carter, G. T., and Servais, LJP

Subjects
Orphan Drug Production, Duchenne muscular dystrophy, Pharmacy, Model-informed drug development, 030226 pharmacology & pharmacy, Models, Biological, Food and drug administration, 03 medical and health sciences, 0302 clinical medicine, Models, medicine, Humans, Regulatory science, Computer Simulation, Muscular Dystrophy, Drug development tools, Duchenne muscular dystrophy consortium (D-RSC), Rare diseases, Regulatory endorsement, Clinical Trials as Topic, Muscular Dystrophy, Duchenne, United States, United States Food and Drug Administration, Pharmacology, Protocol (science), business.industry, Duchenne, Biological, medicine.disease, Clinical trial, Risk analysis (engineering), Drug development, 030220 oncology & carcinogenesis, Aggregate data, Business
Abstract

Drug development for rare diseases is challenged by small populations and limited data. This makes development of clinical trial protocols difficult and contributes to the uncertainty around whether or not a potential therapy is efficacious. The use of data standards to aggregate data from multiple sources, and the use of such integrated databases to develop statistical models can inform protocol development and reduce the risks in developing new therapies. Achieving regulatory endorsement of such models through defined pathways at the US Food and Drug Administration and European Medicines Authority allows such tools to be used by the drug development community for defined contexts of use without further need for discussion of the underlying model(s). The Duchenne Regulatory Science Consortium (D-RSC) has brought together multiple stakeholders to develop a clinical trial simulation tool for Duchenne muscular dystrophy using such an approach. Here we describe the work of D-RSC as an example of how such an approach may be effective at reducing uncertainty in drug development for rare diseases, and thus bringing effective therapies to patients faster.

Published
2019

22. G.P.99 : The Wilmington Robotic Exoskeleton (WREX) improves upper extremity function in patients with Duchenne muscular dystrophy.

Author

Estilow, T., Glanzman, A., Flickinger, J., Powers, K.M., Moll, A., Medne, L., Tennekoon, G., and Yum, S.W.

Subjects
*ROBOTIC exoskeletons, *ARM physiology, *DUCHENNE muscular dystrophy, *AMBULATORY patient groups, *MEDICAL technology, *PSYCHOSOCIAL factors, *WELL-being, *PATIENTS
Abstract

Non-ambulatory patients with Duchenne muscular dystrophy (DMD) have arm weakness limiting anti-gravity movement resulting in severe functional impairments. The Wilmington Robotic Exoskeleton (WREX), by employing a unique 4 bar link technology married with a variable linear elastic band power source, allows for 3 dimensional gravity reduced movement of the arms to enhance function for the completion of Activities of Daily Living (ADL). To establish the utility of WREX in patients with DMD, we report our experience from 2011 to 2014. Nine boys with DMD, aged 11–17y/o (median age 16y/o), were evaluated. Active range of motion (AROM) and functional task completion (self-feeding, drinking from a glass, access to face for grooming, manage eyeglasses, and access their environment, computer, cell phone, and other technology) were completed both with and without the WREX. AROM (without WREX/with WREX) was the following: shoulder flexion 0–30 degrees/45–100 degrees, shoulder abduction 0–30 degrees/45–100 degrees, and elbow flexion was 0–100 degrees/80–120 degrees. With the WREX, all patients demonstrated increased functional skills, especially the ability to self-feed, access joystick, access face for grooming, and reach away from the body to use technology. We conclude WREX provides a decreased gravity environment that allows for significant increased AROM at the shoulder and elbow joints. In addition, WREX allows for the patients to reach away from the body and across midline, improved independence with ADL completion, as well as increased safety in the home and community. WREX may also improve psychosocial wellbeing as all patients demonstrated a positive emotional response to WREX use. [ABSTRACT FROM AUTHOR]

Published
2014
Full Text
View/download PDF

24. P.48 - Should motor function determine the timing of scoliosis surgery in spinal muscular atrophy?

Author

Dunaway Young, S., Montes, J., Salazar, R., Glanzman, A., Pasternak, A., Quigley, J., Ciurylo, E., Riley, S., Martens, W., Gee, R., Duong, T., Civitello, M., Chiriboga, C., Tennekoon, G., Day, J., Finkel, R., Darras, B., and De Vivo, D.

Subjects
*SPINAL muscular atrophy, *SCOLIOSIS treatment, *SPINAL surgery, *MOTOR ability, *ASTHENIA, *MEDICAL decision making
Published
2016
Full Text
View/download PDF

25. G.P.227 - Genetic polymorphisms modify intramuscular fat infiltration in Duchenne muscular dystrophy.

Author

Barnard, A., Romero de Mello Sa, S., Willcocks, R., Senesac, C., Finkel, R., Forbes, S., Sweeney, H., Tennekoon, G., Triplett, W., Lott, D., Wang, D., Byrne, B., Hammers, D., Pham, J., Rooney, W., Finanger, E., Walter, G., Vandenborne, K., and Russman, B.

Subjects
*DUCHENNE muscular dystrophy, *MUSCLE weakness, *MUSCLE abnormalities, *POPULATION genetics, *GENETIC polymorphisms
Published
2015
Full Text
View/download PDF

Catalog

Books, media, physical & digital resources
See catalog results