Update: Motor Outcomes to Validate Evaluations in FSHD

Report by Dr. Statland
See also Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

The primary goal of our Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) study is to hasten therapeutic development and improve care delivery for FSHD by determining the predictive value of clinical characteristics and motor assessments on health outcomes (i.e., need for orthotics, ambulation aides, respiratory support, etc.). The purpose of this study is to understand functional motor assessments, define which assessments are meaningful to people with FSHD, and if certain values of outcome assessments help predict future motor or health outcomes. MOVE FSHD is also unique in that it is truly meant to include all individuals with FSHD. Including those with FSHD type 2, children, and those who are in a wheelchair.

We have enrolled almost 300 participants, and more than half of study participants have come back for annual follow-up visits!  The study continues to enroll new participants. We are also working to add new study sites in Canada and the United Kingdom. This large study will be a big achievement in the FSHD community, a reality that has been made possible by the support from Friends of FSH Research.  Additionally, the MOVE FSHD study has also attracted the notice of industry and other funders – and we were able to increase number of FSHD participants to 470 and add the MOVE+ sub-study that includes whole-body MRI and additional biomarkers, which are essential to accelerate future FSHD drug development. Finally, we plan to formally expand and increase the number of pediatric FSHD participants in the MOVE study.