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Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome

Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome
Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Genethon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of the Liver) annual International Liver Congress on June 26. Based on initial observations, the drug candidate is well tolerated and the first therapeutic effects have been demonstrated, to be confirmed as the trial continues.
Crigler-Najjar syndrome is a rare genetic liver disease characterized by abnormally high levels of bilirubin in the blood (hyperbilirubinemia). This accumulation of bilirubin is caused by a deficiency of the UGT1A1 enzyme, responsible for transforming bilirubin into a substance that can be eliminated by the body, and can result in significant neurological damage and death if not treated quickly. At present, patients must undergo phototherapy for up to 12 hours ....

Giuseppe Ronzitti , Crigler Najjar , Lorenzo Dantiga , Frederic Revah , Emily Henderson , International Liver Congress On , European Association , International Liver Congress , Lorenzod Antiga , Azienda Ospedaliera Papa Giovanni , Liver Gene Therapy , எமிலி ஹென்டர்சன் , ஐரோப்பிய சங்கம் , சர்வதேச கல்லீரல் காங்கிரஸ் ,

Preliminary results of clinical trial for Crigler-Najjar syndrome

Preliminary results of clinical trial for Crigler-Najjar syndrome
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France General , Noord Holland , Giuseppe Ronzitti , Crigler Najjar , Lorenzo Dantiga , Azienda Ospedaliera Papa Giovanni , Frederic Revah , Academic Medical Center , European Union , International Liver Congress On , European Association , International Liver Congress , Lorenzod Antiga , Liver Gene Therapy , Ospedaliera Papa Giovanni , Medical Center , நூற்த் ஹாலண்ட் , ஐரோப்பிய தொழிற்சங்கம் , ஐரோப்பிய சங்கம் , சர்வதேச கல்லீரல் காங்கிரஸ் , மருத்துவ மையம் ,