PURPOSE OF REVIEW: This review aims to describe the benefits and limitations of 
using the Duchenne Connect patient registry to provide information particularly 
in regard to active treatment choices in Duchenne muscular dystrophy and their 
impact on disease progression.
RECENT FINDINGS: Clinical trials and natural history studies are difficult for 
rare diseases like Duchenne muscular dystrophy. Using an online patient 
self-report survey model, Duchenne Connect provides relevant data that are 
difficult to gather in other ways. Validation of the overall dataset is 
supported by comparable mutational spectrum relative to other cohorts and 
demonstrated beneficial effect of corticosteroid use in prolonging ambulation. 
These types of analyses are provocative and allow multivariate analyses across 
the breadth of patient and physician medication and supplement practices. 
Because the data are self-reported and online, the barrier to participation is 
low and great potential exists for novel directions of further research in a 
highly participatory forum.
SUMMARY: Patient registries for Duchenne and Becker muscular dystrophy (DBMD) 
are powerful tools for monitoring patient outcomes, comparing treatment options, 
and relating information between patients, researchers, and clinicians. Duchenne 
Connect is an online patient self-report registry for individuals with DBMD that 
facilitates aggregation of treatment modalities, outcomes, and genotype data and 
has played a vital role in furthering DBMD research, particularly in the USA, in 
a highly participatory and low-cost manner.