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Patient-Focused Clinical Trial Design for Rare Diseases – Part 2

In our previous post, we discussed how patient-focused clinical trial design and collaborative approaches to site enrollment can accelerate rare disease clinical research. Yet, as our presenters emphasized in the presentation, the only way to ensure that trials are being conducted in a truly-patient focused way is to hear directly from rare disease patients and their families. In this post we summarize the insights from Beth Woelfel Harvey, a parent of a child with Duchenne muscular dystrophy (DMD).  

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Patient-Focused Clinical Trial Design for Rare Diseases – Part 1

A few months ago, Medpace and the Cincinnati Children’s Hospital Medical Center presented a webinar on the importance of patient-centric clinical research. Over the next several weeks, we will build off each topic that was addressed during the presentation. In this post, we take a look at what patient-centric clinical trial design looks like for these studies.

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Duchenne Muscular Dystrophy: Challenges in Clinical Research

Duchenne Muscular Dystrophy (DMD) is a specific type of muscular dystrophy characterized by muscle weakness and deterioration. DMD research comes with its fair share of challenges, especially with pediatric trials. We’ll go into a few of these challenges and how Medpace is positioned to overcome them.

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