MDA 2021 poster: Propensity score-matching enables comparison of rare disease product registry data with an external study comparator
This poster, presented at the 2021 virtual MDA Conference, shows the methodology and importance of propensity score-matching in clinical trials where random assignment to treatment is not possible
Review the rationale for using propensity score-matching of registry data as a way to determine the long-term effectiveness of treatments for rare diseases
Explore the methods for identifying propensity score-matched populations to determine safety and efficacy of a mutation-specific treatment for Duchenne muscular dystrophy (DMD)
Understand why the demographics of both populations from the STRIDE and CINRG DNHS registries are presented as a working comparison
▼ 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-2200031 | March 2022
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