Advancing Craniosynostosis Treatment (ACT) Rare Diseases Consortium: Pilot Study Program (LOI)

The Program is designed to support early-stage, high-impact pilot projects that generate preliminary data, validate novel concepts, or de-risk innovative approaches with strong potential for future translational, clinical, or commercialization impact. Proposed projects should align with the mission of the ACT Rare Diseases Consortium and complement or expand the Consortium’s ongoing Clinical Projects. Clinical Project 1 aims to study the longitudinal natural history of syndromic CS. Clinical Project 2 aims to study surgical treatment outcomes of syndromic CS after cranial vault remodeling. Clinical Project 3 aims to study surgical treatment outcomes of syndromic CS after midface advancement.

Responsive topics include, but are not limited to:

* Disease mechanisms, pathophysiology, or target identification

* Biomarker discovery or validation

* Therapeutic development (e.g., small molecules, biologics, cell or gene therapies, and medical devices)

* Diagnostic or prognostic tools

* Data science, artificial intelligence/machine learning, and digital health approaches

Letters of Intent/Inquiry should be limited to one page.

Funding: $40,000 for one year.

https://grants.rarediseasesnetwork.org/grants/ACT-Cycle2-LOI

https://act.rarediseasesnetwork.org/pilot-study-program