The FSHD Society, SOLVE FSHD, and the FSHD CTRN Announce Launch of Industry Collaborative to Improve FSHD Clinical Trials

The FSHD Industry Collaborative is a first-of-its-kind, multi-stakeholder initiative designed to improve how FSHD clinical trials are designed, executed, and evaluated

RANDOLPH, Mass. and VANCOUVER, BC, April 8, 2026 /PRNewswire/ -- The FSHD Society, a patient-driven organization advancing research and clinical development in facioscapulohumeral muscular dystrophy (FSHD), SOLVE FSHD, a venture philanthropic organization dedicated to accelerating treatments for FSHD, and the FSHD Clinical Trial Research Network (FSHD CTRN), a large academic consortium validating biomarkers and outcome measures in FSHD, today announced the launch of the FSHD Industry Collaborative project.

Jointly led by the FSHD Society, SOLVE FSHD, and FSHD CTRN, the Industry Collaborative brings together industry experts in drug development, academic experts, technology partners, and funders to analyze biomarker, natural history, and clinical endpoint data and develop a platform for analyzing patient and clinical trial data for FSHD. The goals are to generate datasets and actionable insights that reduce clinical development risk, improve patient selection hypotheses, improve the understanding and use of clinical measures, and support a more unified regulatory narrative for FSHD programs.

"SOLVE FSHD is pleased to provide foundational funding support to launch this Industry Collaborative and advance biomarker development in FSHD," said Eva Chin, Executive Director, SOLVE FSHD. "By combining natural history data and existing clinical trial data, and best-in-class clinical data partner input, we are building the tools and infrastructure needed to reduce drug development risk and accelerate meaningful progress for the FSHD community."

Turning Data into Field-Wide Learning

Unlike traditional single-sponsor data analyses, the collaboration is structured as a pre-competitive effort to broaden the view across multiple data sets. The Collaborative operates under structured governance to enable cross-program learning and the standardization of clinical tools for FSHD while protecting sponsor confidentiality. Activities include:

• New analysis of donated clinical trial data and natural history datasets to evaluate endpoint performance, variability drivers, and the impacts of patient selection criteria
  
  
• Development and validation of FSHD biomarker assays using patient samples, which can be linked to imaging, patient-reported, and functional outcomes data

Partnering with Expert Stakeholders to Answer Key Strategic Questions

The FSHD Society, SOLVE FSHD, and FSHD CTRN recognize the importance of multiple contributors to this project, reflecting a shared commitment to accelerating the field.

The FSHD CTRN provides expert advisors and highly valuable longitudinal patient data from across more than 30 academic centers and 10 countries with robust expertise in FSHD clinical trials and natural history studies. Biopharma companies Sanofi and Fulcrum Therapeutics will also support the Collaborative, and Fulcrum Therapeutics generously donated clinical data from the Phase 2 and Phase 3 trials of losmapimod to the FSHD Society after the program was suspended in 2024.

Founding industry sponsors of the Collaborative include Scholar Rock, a global biopharmaceutical company developing apitegromab for the treatment of patients with neuromuscular diseases, including FSHD. Several additional drug sponsors are in advanced stages of formalizing their participation, and initial Collaborative work is now underway.

The Global FSHD Innovation Hub, a wholly owned subsidiary of the FSHD Society, will provide strategic advisory, project and alliance management, and contracting services for the Collaborative. Best-in-class technology partners have been engaged to conduct regulatory-ready data analyses and provide FSHD biomarker expertise, including AMRA Medical, Immunologix Laboratories, and Springbok Analytics. Analysis Group, a consulting firm with decades of experience in evidence generation for drug development and regulatory evaluation, will conduct rigorous, collaborative analysis of these complex datasets to advance understanding of disease progression and endpoints, inform future clinical development programs, and help maximize patient benefit from these valuable data.

Building on Prior FDA Engagement

The project will generate data that address outcomes from the FSHD Society and FSHD CTRN's prior engagements with the U.S. Food and Drug Administration, including a 2019 Industry Collaborative Workshop which identified obstacles to therapeutic development, a 2020 EL-PFDD Voice of the Patient Forum describing the lived experience and critical unmet needs in FSHD, a 2024 Patient Listening Session on upper-body mobility and desired treatment outcomes, and a 2025 Adjunct Scientific Workshop in which FSHD patients, clinicians, and scientific experts discussed the importance of clinically meaningful and sensitive endpoints in FSHD trials. Several experts who participated in these engagements are contributors to the Industry Collaborative.

"Aligning patient experience, scientific rigor, and regulatory expectations is essential for progress in FSHD," said Dr. Lucienne Ronco, CSO, FSHD Society. "This initiative reflects the natural next step following our FDA engagements to the data-driven execution of these important projects."

Open Invitation During Founding Phase

The Collaborative is welcoming additional pharmaceutical and biotech company sponsors, technology partners, donors, and mission-aligned investors to participate.

Community donations and philanthropic capital will directly support the completion and expansion of the work, accelerating the generation of insights intended to benefit both the FSHD community and ongoing clinical development programs.

"Access to high-quality clinical trial and natural history data is critical for improving trial design for rare diseases," said Amanda Rickard, Managing Director, Global FSHD Innovation Hub, and Collaborative Director. "This research collaboration creates a rigorous framework for generating insights that individual FSHD programs cannot achieve in isolation."

SOURCE FSHD Society