The Power is in Your Hands: Duchenne Regulatory Science Consortium

About a month ago, I wrote to you to announce the launch of a new partnership between PPMD and the Critical Path Institute (C-Path) called the Duchenne Regulatory Sciences Consortium or D-RSC (see “Different Together: PPMD Announces the Duchenne Regulatory Science ...”).  I’m writing today to tell you a bit more about D-RSC, tell you about an important milestone, and to ask for your help.

 

D-RSC is an effort to bring together de-identified data from as many sources as possible in order to understand the variability that we see in Duchenne, and to develop a Duchenne progression model.  Once developed, this model will provide predictive value to the design of clinical trials—meaning that we will be able to do better trials, faster trials, and trials that minimize (and possibly eliminate) placebo controls. 

 

Together we can do this

Like everything we do at PPMD, we’re being ambitious in the D-RSC effort and have recruited academics, companies, and the NIH and FDA as consortium partners. Make no mistake, the goal of a Duchenne progression model will not happen unless we aggregate data, your data, from a wide breadth of clinical studies at universities and research institutes, as well as clinical trial data from companies.  

 

Repeat—no company or academic group can achieve this goal on their own; only by working together as a community of physicians, clinical scientists, drug developers, families, and people living with Duchenne can we accomplish this goal. 

 

This will require that we bring together as much data on as many different aspects of Duchenne as we can for this model. This model will benefit any therapy development effort, by any company, for any dystrophin mutation.  Like PPMD’s mission, we’re an all-inclusive partnership!

 

We need your help

In terms of an update on D-RSC, you should know that DuchenneConnect data, your data*, is the first to be transferred to the Critical Path Institute for development of the progression model.  More datasets from academics and industry will soon become part of the model development.  So:

 

  • If you already participate in Duchenne Connect, congratulations! The only thing we ask we is to make sure your account is updated  now.
  • If you haven’t joined, join now and help us build this dataset while helping yourself to a vast educational experience.

 

In either case, take the time to explore what DuchenneConnect has to offer, while helping us achieve the critically important goals of D-RSC.  DuchenneConnect is a one-of-a-kind resource that is now helping to drive the ambitious goals of D-RSC – your information is fostering a critically important research goal. 

 

Collaboration is an incredibly powerful weapon in our fight to end Duchenne and we are proud of the way D-RSC is illustrating such teamwork. But we can’t do it without your participation in programs like DuchenneConnect…the power is in your hands!

 

*Remember that your privacy is very important to us and when your data in DuchenneConnect is shared, it is always de-identified.  

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