Seeking Novel Solutions to Treat Rare Genetic Skeletal Muscle Diseases

Our client, a global leader in therapies for rare genetic diseases, is looking for novel approaches to target the primary cause of rare genetic skeletal muscle diseases, to transform the way patients feel, function, and survive.

The following conditions are of interest: Muscular Dystrophies (Duchenne, Becker, FSHD, Limb-girdle, LAMA2, Emery-Dreifus, Ullrich, Oculopharyngeal) Myotonic Dystrophy Type 1 and 2, Pompe Disease, Nemaline Myopathy, X-linked Centronuclear Myopathy, RYR1-related myopathies and other rare genetic skeletal muscle diseases will be considered...