An observational study building a patient data repository that provides real world experience regarding the use of therapeutic treatments in clinical practice.
What does STRIDE mean?
STRIDE stands for 'Strategic Targeting of Registries and International Database of Excellence.' The STRIDE registry has been set up in neuromuscular disorders (NMD) within a STRIDE-NMD umbrella registry concept that will enable a broader disease area registry to be created.
What is the purpose of the registry?
The discovery and development of novel therapeutic agents, especially for rare diseases does not end with the completion of clinical trials required for registration and marketing. The STRIDE-NMD patient registry will extend the evidence base into clinical practice, thus securing a broader understanding of real world safety, effectiveness and usage of novel therapeutic treatments. Regulatory authorities including the EMA have begun to execute this registry as part of the post marketing approval commitment.
How will it be run?
The first STRIDE-NMD patient registry is a collaborative partnership between TREAT-NMD and PTC Therapeutics. The registry will be led by a Steering Committee of leading DMD specialists, patient advocates and PTC representatives.
- The Committee will oversee the interrogation of the repository of patient-registry data by establishing Working Groups consisting of experts to define and lead clinically relevant research topics
- Participating investigators will be invited to propose research and take part in or lead these Working Groups