Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Publications | Publication: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy (DMD) patients

Publication: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy (DMD) patients

This research article, published in the Journal of Comparative Effectiveness Research, provides results from a study investigating the efficacy of a mutation-specific therapy in delaying loss of ambulation (LoA) and respiratory function in patients with nonsense mutation DMD (nmDMD)

Learn about the demographics of patients enrolled in the long-term Phase 3 propensity score-matched study
Review the data on age at LoA in the treated vs untreated populations
Understand the decline in lung function, as measured by reduction in predicted forced vital capacity to <60% across the 2 groups

▼ 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-2200033 | June 2025

Publication: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy (DMD) patients

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