Do you ever wonder what happens to all the data we collect in the DuchenneConnect Registry? Ever wonder why we regularly ask you to update your registry account?

How your data is used:

Your data is used in many ways. Frequently researchers working in the pharmaceutical industry request the DuchenneConnect data to help them with planning their clinical trials.  And sometimes researchers or clinicians specializing in Duchenne request specific registry data to help them answer an important question.  A few years ago we were approached by Dr. Richard Finkel, a neurologist at Nemours Children's Hospital in Orlando, Florida. He and his team wanted to determine if the age at onset of ambulation (age at first steps) was significantly later for toddlers with Duchenne compared to toddlers without Duchenne. We created a survey with questions focused on this topic (First Steps/Walking Module) and almost 500 registrants completed this survey!

New publication suggests that age at onset of ambulation could be a primary outcome measure in clinical trials involving treatment in early infancy:

Dr. Finkel and his team analyzed the data from the DuchenneConnect survey, as well as data from MD STARnet and the Dutch Natural History Survey.  Their completed manuscript was recently published in Neuromuscular Disorders. They found that the age at onset of ambulation (age at first steps) is significantly delayed in boys with Duchenne. They suggest that age at onset of ambulation could be a primary outcome measure in clinical trials involving treatment in early infancy.  This is important because the most commonly used outcome measure in clinical trials is the 6-minute walk test, but that is impossible for infants to perform. If we could use the age at onset of ambulation as the primary outcome measure in a clinical trial, we could potentially have more treatment trials that include infants with Duchenne.

Thank you to all the families that completed the First steps/Walking Module in the past. Your time and effort are greatly appreciated! 

And thank you for joining the fight to End Duchenne!

If you would like to register on DuchenneConnect or update your Medical History surveys, please go to DuchenneConnect.org!  

Questions? 

Call 888-520-8675 or email coordinator@duchenneconnect.org.