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Goal: The ACT Rare Diseases Consortium Pilot Study 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.
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