Posted by George Shaw on May 25, 2018
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As potential therapeutics come down the pipeline, we do not want to only then begin to consider clinical trials, and so both Drs. Jeff Statland and Rabi Tawil have taken the lead on this. The provided grant not only allows for expansion of the clinical trials network, but also producing the important outcome measures. That is, how does one know if a potential therapeutic is both effective and makes a difference for the patient? It is not as easy as one would think, especially in such a highly variable disease as FSHD.
The study, Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD, is described on ClinicalTrials.gov, and contains inclusion criteria and contact information at each site for those interested in particpating.
Note that this grant and study is one of the outcomes of the 2016 ENMC conference (see article and travel grant).
For more information on patient-reported outcomes, see the March 2018 webinar conducted by FasterCures Patient-Centered Measurement: How Close Are We?
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