| | Our efforts have yielded promising results, with some of our early-phase clinical trials leading to later-stage research and FDA-approved breakthroughs. A notable example is the development of a drug to treat pediatric patients with spinal muscular atrophy. However, we need to do more and expand our efforts to provide research access to advanced therapeutics to more children. To this end, we have strategic plans to increase the number of early-phase studies we conduct, backed by our commitment to invest in our investigators, staff, and resources to strengthen our clinical research infrastructure. We do this with our commitment to equitable and diverse access to our clinical trials. We believe that diversity in our trials enhances the generalizability of our findings, promotes health equity, and reduces health disparities. Our goal is to make our trials more accessible and welcoming to diverse populations, thereby working toward reducing healthcare inequities and improving health outcomes for all children.
Guiding this evolution are Ashley Hoambrecker, Senior Director of the Office of Clinical and Community Trials, and Bill Muller, MD, PhD, Scientific Director of the Clinical and Community Trials Pillar. They believe that fostering a research culture across Lurie Children’s is crucial in translating the ideas of our basic scientists into clinical interventions. Their approach includes strategies like creating clinical research support units within our Clinical Research Services line.
Their vision is to create a structure that encourages all our clinicians, even those with limited experience in clinical research, to explore cutting-edge therapies. This approach, coupled with institutional systems that allow expertise to expand across disciplines, positions Manne Research Institute as a desirable location for early-phase trials in partnership with industry, particularly for smaller companies with limited resources.
Our commitment to providing advanced therapeutics to critically ill children has never been more crucial. Building out a world-class first-in-child early-phase clinical trials unit will provide patients with limited options access to cutting-edge therapies. To be a pediatric hospital and research institute enterprise that consistently provides new options to our most critically ill patients with complex diagnoses is to give hope to the families that there might be something better for their child and children in the future.
With kindness and respect,
Pat
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