AAN 2021 poster: Ataluren delays loss of ambulation and decline in pulmonary function in patients with nonsense mutation Duchenne muscular dystrophy
This poster, from the 2021 virtual AAN meeting, provides results from a study evaluating the efficacy of a mutation-specific treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD)
Explore the background, methods, and results of a propensity score-matched post hoc registry study to investigate the safety and efficacy of a mutation-specific treatment for nmDMD
Review the characteristics of patients, who received either a mutation-specific treatment for nmDMD + standard of care (SoC), or SoC alone
Learn about the Kaplan-Meier analyses of patient age at loss of ambulation and age at decline in pulmonary function, measured by predicted forced vital capacity
▼ 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 email@example.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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MED-ALL-DMD-2200031 | March 2022
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