Ataluren truly is a product and a pipeline. Ataluren has demonstrated activity in multiple nonsense mutation cell and animal-based disease models in different organ systems and therefore has the potential to treat multiple rare disorders. Below are over 20 documented examples of preclinical studies that demonstrate ataluren's activity in multiple different nonsense mutation cell and animal-based disease models in different organ systems.

General Ataluren Publications

Muscle Disorders

Ion Channel Disease

Neurological Disorders

Skin Disease

Eye Disorders

Pulmonary Disease

Metabolic Disorders

Reporter Assays

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