Rare diseases
Investigational molecules
Locations
1 Rue De L Internationale, 91000 Evry-Courcouronnes, France
Clinical research includes Crigler-Najjar syndrome and severe Crigler-Najjar syndrome in patients requiring phototherapy, with a focus on restoring bilirubin metabolism and addressing persistent hyperbilirubinemia.
Work in this area centers on liver-directed gene therapy and long-term clinical assessment of metabolic correction.
Genethon runs clinical trials in conditions such as Crigler-Najjar syndrome, Wiskott-Aldrich syndrome, Duchenne muscular dystrophy, Chronic granulomatous disease, among others.
Clinical trials run by this sponsor – those enrolling participants first.
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