There are currently no effective treatments to halt the muscle breakdown in Duchenne muscular dystrophy (DMD), although genetic-based clinical trials are being piloted. Most of these trials have as an endpoint the restoration of dystrophin in muscle fibers, hence requiring sufficiently well-preserved muscle of recruited patients. The choice of the muscles to be studied and the role of noninvasive methods to assess muscle preservation therefore require further evaluation.

Kinali, M., Arechavala Gomeza, V., Cirak, S., Glover, A., Guglieri, M., Feng, L., Hollingsworth, K., Hunt, D., Jungbluth, H., Roper, H., Quinlivan, R., Gosalakkal, J., Jayawant, S., Nadeau, A., Hughes Carre, L., Manzur, A., Mercuri, E. M., Morgan, J., Straub, V., Bushby, K., Sewry, C., Rutherford, M., Muntoni, F., Muscle histology vs MRI in Duchenne muscular dystrophy, <<NEUROLOGY>>, 2011; 76 (4): 346-353. [doi:10.1212/WNL.0b013e318208811f] [http://hdl.handle.net/10807/6747]

Muscle histology vs MRI in Duchenne muscular dystrophy

Mercuri, Eugenio Maria;
2011

Abstract

There are currently no effective treatments to halt the muscle breakdown in Duchenne muscular dystrophy (DMD), although genetic-based clinical trials are being piloted. Most of these trials have as an endpoint the restoration of dystrophin in muscle fibers, hence requiring sufficiently well-preserved muscle of recruited patients. The choice of the muscles to be studied and the role of noninvasive methods to assess muscle preservation therefore require further evaluation.
2011
Inglese
Kinali, M., Arechavala Gomeza, V., Cirak, S., Glover, A., Guglieri, M., Feng, L., Hollingsworth, K., Hunt, D., Jungbluth, H., Roper, H., Quinlivan, R., Gosalakkal, J., Jayawant, S., Nadeau, A., Hughes Carre, L., Manzur, A., Mercuri, E. M., Morgan, J., Straub, V., Bushby, K., Sewry, C., Rutherford, M., Muntoni, F., Muscle histology vs MRI in Duchenne muscular dystrophy, <<NEUROLOGY>>, 2011; 76 (4): 346-353. [doi:10.1212/WNL.0b013e318208811f] [http://hdl.handle.net/10807/6747]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/6747
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