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Publication: Early treatment with ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy

This case study, published in Acta Myologica, details an ambulant 2-year-old patient with nonsense mutation Duchenne muscular dystrophy (nmDMD), who received early treatment with a mutation-specific therapy in an expanded-access program

Read about the background, clinical presentation, and diagnosis of the patient, who began treatment with a mutation-specific therapy at 2 years of age
Review the clinical outcomes of early treatment with a mutation-specific therapy for nmDMD over 16 months, as measured by assessment of the patient’s motor, cognitive, pulmonary, and cardiac function

▼ Ataluren is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.

MED-ALL-ATLN-2500006 | September 2025

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MED-ALL-DMD-2200118 | June 2025

Publication: Early treatment with ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy

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