Duchenne Muscular Dystrophy: Genethon's Gene Therapy Enters Final Phase of Trial
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Genethon must find the necessary funding to complete gene therapy research, which will cost around €115 million. Here, during the design and development of drugs in the Genethon laboratories in Évry-Courcouronnes, in November 2024. Thierry Thorel / PhotoPQR/Voix du Nord/MaxPPP
On Friday, July 25, the Généthon laboratory received authorization from the European Medicines Agency to begin phase III trials of its gene therapy. This is a crucial step before a possible market launch in five years.
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I subscribeSuffering from Duchenne muscular dystrophy, a genetic disease that progressively destroys muscles, little Sacha, 8, was doomed to a wheelchair. But nearly three years after receiving the treatment developed by the Généthon laboratory, the young boy is bursting with vitality. "Seeing him get up to mischief is a real joy," his parents, Hélène Wilhelm and Édouard Villemin, rejoice.
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