Myotonic Muscular Dystrophy

Myotonic Muscular Dystrophy

IPLEX was investigated in a Phase II clinical study at the University of Rochester School of Medicine, with funding provided by the Muscular Dystrophy Association and the National Institutes of Health. This Phase II program studied the safety and tolerability of once-daily, subcutaneous injection of IPLEX in patients with MMD. While patients with MMD showed significant increases in total muscle weight, testosterone levels, and LDL levels, and significant decreases in triglyceride and HDL levels, functional assays such as grip strength and walk tests did not show improvement. Muscular dystrophy (MD) is a group of muscle diseases that weaken the musculoskeletal system and hamper locomotion. Muscular dystrophies are characterized by progressive skeletal muscle weakness, defects in muscle proteins, and the death of muscle cells and tissue. In the 1860s, descriptions of boys who grew progressively weaker, lost the ability to walk, and died at an early age became more prominent in medical journals. In the following decade, French neurologist Guillaume Duchenne gave a comprehensive account of thirteen boys with the most common and severe form of the disease, which now carries his name—Duchenne muscular dystrophy. Myotonic dystrophy (dystrophia myotonica, myotonia atrophica) is a chronic, slowly progressing, highly variable, inherited multisystemic disease. 

Myotonic Muscular Dystrophy

Myotonic Muscular Dystrophy

Myotonic Muscular Dystrophy

Myotonic Muscular Dystrophy

Myotonic Muscular Dystrophy

Myotonic Muscular Dystrophy

Myotonic Muscular Dystrophy

Myotonic Muscular Dystrophy

Myotonic Muscular Dystrophy