Last week, Mark Stone, the FSHD Society president and CEO, gave his fourth-quarter progress report on the Therapeutic Accelerator for FSHD treatments. His presentation was given via webinar to leadership donors and key volunteers, including chapter directors and Walk & Roll leaders. The recorded talk and Q&A are now available on the FSHD Society’s YouTube channel. (Click above to view.)
Stone summarized findings from the FSHD Society’s Industry Collaborative Workshop held in March, 2019, which engaged academic leaders, industry, and FDA representatives, in a day-long discussion of clinical trial preparedness for FSHD. The workshop identified key gaps in genetic testing and in methods to measure disease progression through the use of blood biomarkers, imaging technology, and analysis of data from FSHD patient registries around the world. Working with academic and industry partners, the FSHD Society has been mapping out specific projects to overcome these gaps, and is launching an alliance to collaborate in funding and carrying out the necessary work.
Stone also disclosed that the FSHD Society had received approval from the US Food and Drug Administration to hold an externally led Patient-Focused Drug Development meeting, now scheduled for April 21, 2020.
The CEO progress report also detailed the explosive growth in community engagement, through the nationwide chapter program which now numbers 26 local chapters. The FSHD Society’s signature Walk & Roll fundraiser has raised more than $400,000 this year, contributing significantly to the war chest that will be deployed on the Therapeutic Accelerator.