Ataluren

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Ataluren

Ataluren (pronounced as: ah-TAL-uh-ren) is a pharmaceutical drug used in the treatment of Duchenne muscular dystrophy (DMD) and nonsense mutation cystic fibrosis. It is marketed under the trade name Translarna.

Etymology

The name "Ataluren" is derived from the phrase "ataluren the nonsense", which refers to the drug's mechanism of action in overcoming nonsense mutations in genetic disorders.

Mechanism of Action

Ataluren works by allowing the cell's protein-making machinery to skip over the mutation, enabling the production of a functional protein. This process is known as read-through.

Usage

Ataluren is used in the treatment of Duchenne muscular dystrophy and cystic fibrosis caused by nonsense mutations. It is taken orally, usually three times a day.

Side Effects

Common side effects of Ataluren include headache, nausea, vomiting, and fatigue. More serious side effects may include kidney damage.

Related Terms

External links

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