CORDIS Project
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This project aims to advance the clinical development of nitisinone for treating Alkaptonuria, a rare genetic disorder. It will involve studies to determine the drug's efficacy and the optimal timing for treatment, addressing a significant unmet medical need.
DevelopAKUre is a proposal to fund the clinical development of an orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). AKU is a genetic deficiency of homogentisic acid dioxygenase, causing high levels of homogentisic acid (HGA).
Oxidation of HGA to pigment polymer, termed ochronosis, alters connective tissues.
This leads to multisystemic damage dominated by premature severe arthritis.
Currently, multiple arthroplasty is inevitable since AKU is in…
ROYAL LIVERPOOL AND BROADGREEN UNIVERSITY HOSPITALS NHS TRUST
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
United Kingdom, Liverpool
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Slovakia, PIESTANY
Type: Public body
Activity type: Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
SME: No
ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France, Paris
Type: Research institute
Activity type: Research Organisations
SME: No
Netherlands, HOOFDDORP
Type: Company (for-profit)
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
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