Ataluren

Ataluren is a small molecule drug developed by PTC Therapeutics and sold under the brand name Translarna in some markets. It is designed to counteract nonsense mutations by promoting ribosomal read-through of premature stop codons, enabling the synthesis of full-length protein that would otherwise be truncated.

It is studied primarily for Duchenne muscular dystrophy caused by nonsense mutations (nmDMD). In the European Union, ataluren has been approved for ambulant patients aged 5 years and older. In the United States, it has not received FDA approval. The drug has also been evaluated in other diseases associated with nonsense mutations, including cystic fibrosis, but did not obtain regulatory approval there.

Evidence from clinical trials has been mixed. Some studies suggested possible stabilization or modest improvement in motor function in certain nmDMD subgroups, while primary endpoints were not consistently met across trials. As a result, efficacy remains a matter of ongoing evaluation and is region-specific.

Safety data from trials report adverse events such as vomiting, fever, cough, and gastrointestinal symptoms; serious adverse events are uncommon. As with any therapy, monitoring for potential liver effects and drug interactions is advised, and dosing should follow regional regulatory labels.