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In a European Phase I/II clinical trial, the experimental drug for treating Crigler-Najjar syndrome reduced bilirubin below toxic threshold in the liver with a single intravenous injection.
Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today announced the appointment of Roger J. Hajjar, MD,
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), today announced that the European Medicines Agency (EMA) has granted PRIME
The gene therapy would represent a first-of-its kind treatment for this life threatening genetic disease Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular
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 abn...