MDA 2022 poster: Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG Natural History Study: A matched cohort analysis

This poster, presented at the 2022 MDA Conference in Nashville, Tennessee, USA, shows results from a propensity score-matched registry analysis, using data from the STRIDE Registry and CINRG Natural History Study, to determine the effect of a mutation-specific treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD) on pulmonary function

• Find out about the demographics of patients in the STRIDE Registry and CINRG Natural History Study who were propensity score-matched to evaluate the effect of a treatment for nmDMD on pulmonary function
• Compare results from 2 cohorts of patients (N=241 in each) who received either a mutation-specific treatment for nmDMD plus standard of care (SoC), or SoC alone
• Review the pulmonary function outcome, as measured by ages at percent predicted forced vital capacity, for each cohort

▼ 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.

Deflazacort is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older. PTC Therapeutics is the Marketing Authorization Holder for deflazacort in the US only. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The US Prescribing Information for deflazacort is available here. Please refer to your local country guidance for more information.

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