Summary
The Congenital Lipomatous Overgrowth, Vascular Malformations, Epidermal Nevi, Scoliosis/Skeletal and Spinal (CLOVES) syndrome is a genetic disorder due to a gain of function mutation of the PIK3CA in a mosaic fashion. This rare condition is associated with severe deformation and poor survival. We identified a new drug able to dramatically improve the outcome of patients with this rare disease. The purpose of the Proof of Concept grant is to better understand the molecular effect of this drug and to rapidly accede to Orphan drug designation.
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More information & hyperlinks
| Web resources: | https://cordis.europa.eu/project/id/737546 |
| Start date: | 01-02-2017 |
| End date: | 31-07-2018 |
| Total budget - Public funding: | 148 750,00 Euro - 148 750,00 Euro |
Cordis data
Original description
The Congenital Lipomatous Overgrowth, Vascular Malformations, Epidermal Nevi, Scoliosis/Skeletal and Spinal (CLOVES) syndrome is a genetic disorder due to a gain of function mutation of the PIK3CA in a mosaic fashion. This rare condition is associated with severe deformation and poor survival. We identified a new drug able to dramatically improve the outcome of patients with this rare disease. The purpose of the Proof of Concept grant is to better understand the molecular effect of this drug and to rapidly accede to Orphan drug designation.Status
CLOSEDCall topic
ERC-PoC-2016Update Date
27-04-2024
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