Novel, point of care biomarker to detect pre-clinical flares for people living with fibrodysplasia ossificans progressiva (FOP)

Awardee: Nezihi Murat Karabacak

Institution: Massachusetts General Hospital

Grant Amount: $64,000

Funding Period: February 1, 2022 - January 31, 2023


Summary:

Fibrodysplasia ossificans progressiva (FOP) is a severely debilitating condition with no accurate and clinically applicable biomarkers or imaging approaches. Our overall goal is to develop a novel, non-invasive assay to detect the initiation and progression of FOP. We will define a series of novel blood-based biomarkers specific for monitoring heterotopic ossification predicated in people living with FOP. As a result, we will establish an innovative way to detect and evaluate progression and monitor treatment outcomes for this disease process.

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Identifying Unmet Care and Communication Needs of Families Affected by Complex Lymphatic Anomalies

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Understanding the role of inflammation in MPSIIIA disease to identify therapeutic approaches for older patients without treatment options