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[THU0369] : THE MEDICAL BURDEN OF RARE AUTOINFLAMMATORY DISEASES: EXEMPLE OF MEVALONATE KINASE DEFICIENCY IN FRANCE

Researchers

Presenter

  • C. Galeotti

Principal Investigators

  • S. Berody

  • I. Kone-Paut

  • M. Piram

Medical Centers

  • Pediatric Rheumatology, CHU Bicetre, Le Kremlin Bicetre, France

Locations

  • France

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

Disease

  • Mevalonate kinase deficiency

  • Inflammatory bowel disease

  • Kawasaki disease

  • Chronic infantile neurologic cutaneous and articular syndrome

  • Lupus

  • Tumor necrosis factor receptor associated periodic syndrome

  • Hyperimmunoglobulinemia D and periodic fever syndrome

Biomarkers

  • Alpha-1-microglobulin/bikunin precursor

Drug/Treatment

  • Hydroxychloroquine

  • Methotrexate

  • Ciclosporine

Outcome

  • N/A

Study Design

  • N/A

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • Interim

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