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Intrathecal onasemnogene abeparvovec (OAV101 IT) demonstrated an increase from baseline in Hammersmith Functional Motor Scale – Expanded total score in children and young adults with SMA.

The top five episodes of our podcast in the year 2024, highlighted.

Study finds that existing patient-reported outcome (PRO) measures fail to fully capture health-related quality-of-life for some conditions, highlighting the need for a novel, disease-specific PRO to better assess treatment efficacy and improve patient care in clinical trials.

Ashley Moultrie, CCRP, senior director, DEI & community engagement, Javara discusses current trends and challenges with achieving greater diversity in clinical trials, how integrated research organizations are bringing care directly to patients, and more.

This model, built around a mix of flexible, empathetic, and results-driven principles, may be uniquely positioned to navigate the changing dynamics and demands for contract development and manufacturing organizations.

The complexities behind these translations require specialized personnel who are nuanced in both medical cultures.