Effects of long-term eteplirsen treatment on upper limb function in patients with Duchenne muscular dystrophy: findings of two phase 2 clinical trials

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Duchenne muscular dystrophy (DMD) is a progressive, degenerative neuromuscular disease caused by gene mutations that result in the absence or deficiency of dystrophin protein. Absence of dystrophin compromises the regenerative ability of muscle fibers, and, as muscle deterioration overtakes muscle growth, loss of upper limb function causes difficulties in performing daily activities and negatively impacts quality of life. Eteplirsen (Exondys 51; Sarepta Therapeutics, Cambridge, MA), the first FDA approved treatment for DMD in the US, is indicated for treatment of patients with DMD amenable to exon 51 skipping.

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