DMD CLINICAL THERAPIES II

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Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, neuromuscular disease caused by DMD gene mutations that disrupt the dystrophin messenger ribonucleic acid (mRNA) reading frame and prevent production of functional dystrophin protein. Eteplirsen is a phosphorodiamidate morpholino oligomer (PMO) approved by US-FDA for treatment of DMD patients with mutations amenable to exon 51 skipping, Eteplirsen excludes exon 51 to restore the dystrophin mRNA reading frame and enable translation of internally shortened dystrophin protein.

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