Our Members

Cure Duchenne logo


About CureDuchenne is recognized as the global leader in research, patient care and innovation for improving and extending the lives of children with Duchenne.
Will individual participant data be available (including data dictionaries)? Yes
What data in particular will be shared? All of the individual participant data collected, after deidentification, for placebo subjects from the phase 2 and 3 randomized, double-blind, placebo-controlled clinical trials of drisapersen for the treatment of Duchenne muscular dystrophy (DMD) as well as observational studies.

The PRO-DMD-01 study was a prospective observational study of disease progression of boys with Duchenne muscular dystrophy.

Exceptions CureDuchenne is only able to approve the use of the data for research purposes at this time and not for use by a commercial organization or as part of a registration package. For-profit entities should contact CureDuchenne directly via monica@cureduchenne.org to verify use and arrangements.
What documents will be available? For clinical trials:

Study data, in a format similar to SDTM

Protocols with all amendments

Annotated case report forms

Dataset specifications (“define” files)

For observation studies:

Raw datasets


Operations manual

For what types of analyses? Investigation of placebo response and the outcomes of comparator (placebo) subjects in DMD clinical trials
By what mechanism will the data be made available? Using the Vivli Platform via secure download