Patient registry for muscular dystrophies to accelerate #Genetherapy development

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Customer: Cure Rare Disease is on a mission to research and develop cost-effective, lifesaving long-term therapies through collaboration with world renowned researchers and clinicians. Led by the Founder, Rich Horgan, inspired by his younger brother with Duchenne muscular dystrophy, is developing a gene therapy for DMD.


The organization was seeking to establish a patient registry and capture patient reported outcomes data for muscular dystrophies. However, the team was struggling with:
  • Identifying how to get started using best practices and tools.
  • How to engage and retain patients suffering from various forms of muscular dystrophies over a prolonged period of time.
  • Finding a cost-effective yet robust solution that is lightweight, easy to use, and mobile-friendly for patients.
  • A requirement of multimodal communications SMS/email/audio/video in one app.
Cure Rare Disease Case Study

Jeeva eCOA Solution

Jeeva’s mobile-friendly eCOA/ePRO solution delivered to Curerd:
  • A BYOD (bring your own device) model for an easy and convenient way for their patients community to engage and participate from the comfort of their own homes, which increased retention and engagement.
  • SMS/email/audio and video solution in one app that provided better bidirectional communications.
  • Over a hundred patients with these rare diseases were engaged and high quality patient reported outcomes data captured in an year.