Translarna™, developed by PTC Therapeutics, is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older. Ataluren is approved within the European Union (EU) Member States, investigational in other jurisdictions.
Ataluren, is a novel, orally administered small-molecule compound for the treatment of patients with genetic disorders due to a nonsense mutation. Ataluren is in clinical development for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation (nmDMD). Ataluren is approved within the European Union (EU) Member States, Iceland, Liechtenstein, Norway, Israel, and South Korea under the trade name Translarna™ for the treatment of nmDMD in ambulatory patients aged five years and older. Translarna is the first treatment approved for the underlying cause of DMD. The European Medicines Agency, or EMA, has designated ataluren as an orphan medicinal product and the U.S. Food and Drug Administration, or FDA, has granted orphan drug designation to ataluren for the treatment of nmDMD.
Duchenne muscular dystrophy can be caused by a number of genetic abnormalities. Translarna is for use in patients whose disease is due to the presence of certain defects (called nonsense mutations) in the dystrophin gene which prematurely stop the production of a normal dystrophin protein, leading to a shortened dystrophin protein that does not function properly. Ataluren works by interacting with the ribosome, which is the component of the cell that decodes the mRNA molecule and manufactures proteins, to enable the ribosome to read through premature nonsense stop signals on mRNA and allow the cell to produce a full-length, functional protein.
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