Video commentary: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy
In this video, recorded in 2022 for the Video Journal of Biomedicine, 2 authors of a research article present the background and findings from their study of a mutation-specific treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD)
In this video, 2 of the study authors, Professor Már Tulinius and Dr Craig McDonald, describe the methodology used in a propensity score-matched study to determine the efficacy and safety of a treatment for nmDMD
Learn about the baseline characteristics of patients who received treatment in this long-term, open-label, Phase 3 study
Listen to the researchers discuss their findings. The primary study objective was to evaluate the safety and effectiveness of the treatment, with efficacy measured by time to disease progression milestones, including age at loss of ambulation, and decline in pulmonary 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 at firstname.lastname@example.org.
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-2200112 | August 2023
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