H. Haddad

A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C.

N. Romero, D. Klatzmann, R. Mulligan, 2012, Brain : a journal of neurology.

Riluzole attenuates spinal muscular atrophy disease progression in a mouse model

J. Melki, C. Cifuentes-Díaz, V. Joshi, 2003, Muscle & nerve.

Arginine butyrate: a therapeutic candidate for Duchenne muscular dystrophy

S. Matecki, F. Fougerousse, L. Bossi, 2013, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.

The CHRNE 1293insG founder mutation is a frequent cause of congenital myasthenia in North Africa

E. Génin, H. Lochmüller, J. Pouget, 2008, Neurology.

congenital myasthenia in North Africa 1293 insG founder mutation is a frequent cause of CHRNE The

E. Génin, J. Pouget, A. Barois, 2008 .

A synonymous CHRNE mutation responsible for an aberrant splicing leading to congenital myasthenic syndrome

B. Echenne, B. Eymard, E. Fournier, 2007, Neuromuscular Disorders.