ICNMD 2021 poster: Pulmonary function in non-ambulatory patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) from the STRIDE ataluren Registry and CINRG Duchenne Natural History Study (CINRG DNHS): A matched cohort analysis

This poster, presented at the ICNMD virtual meeting in 2021, shows findings from a registry study investigating the effects of a mutation-specific Duchenne muscular dystrophy (DMD) therapy on pulmonary function in patients with DMD

  • Understand the background and methods of a study investigating lung function in 2 cohorts of non-ambulatory patients with DMD
  • Explore the results from patients from the STRIDE Registry (treated with a mutation-specific DMD therapy [n=22]), compared with those from propensity score-matched patients in the CINRG DNHS registry (not treated with this mutation-specific therapy for DMD)
  • Review the Kaplan Meier analyses of lung function decline, assessed by predicted forced vital capacity of <60%, <50%, and <1 L

▼ 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 at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for ataluren is available here. Please refer to your local country guidance for more information.

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