Case Study

Early Phase Rescue Study In A Progressive Rare Disease: A Personal Approach

Source: Worldwide Clinical Trials
How One Rare Disease Firm Merged Gene Therapy With Patient Centricity

A Rescue Study

Worldwide oversaw the sponsor’s preceding Phase I study for healthy volunteers and had the infrastructure in place to accommodate a similar study in patients. 

The Challenge

The disorder causes progressive muscle weakness, meaning patients required extra safety considerations and travel accommodations to participate. Because of the health of the patients and the complexity of the therapy, they needed a facility where the study team could monitor patients around the clock. Patients also had to be willing and physically able to travel to San Antonio, Texas, for three weeks for participation.

To learn more about Worldwide Clinical Trials, visit their Clinical Leader page.

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