Givinostat for Duchenne muscular dystrophy

Givinostat is in clinical development for Duchenne muscular dystrophy (DMD). DMD is a genetic
disease that gradually causes weakness and atrophy (wasting) of the muscles. It mainly affects
boys, and usually starts before the age of six years. Patients with DMD lack normal dystrophin, a
protein found in muscles. Because this protein helps to strengthen and protect muscles from
injury as muscles contract and relax, in patients with DMD the muscles become weak and
eventually stop working. DMD causes long-term disability and is life threatening because of its
effects on the heart and the respiratory muscles (muscles that are used to breathe). The disease
usually leads to death in adolescence or early adulthood. Treatment of DMD has been challenged
by the limited number of available medicinal products and the minimal benefits associated with
currently available ones.