News

Genethon wishes you a happy new year 2023!

In 2023, we will not give up and continue the fight against rare diseases

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Publication: Genethon helps clarify a molecular mechanism of mitochondrial malfunction in Duchenne muscular dystrophy

Several laboratories, including Généthon, have begun gene therapy clinical trials for Duchenne muscular dystrophy. Research must be conducted, however, to improve the therapeutic benefit of future treatments for this rare genetic disease. 

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Téléthon 2022: Benjamin, a hope for families thanks to clinical trials

Suffering from Duchenne muscular dystrophy, a neuromuscular disease that is now incurable, Benjamin, 10, is one of the ambassadors of Téléthon 2022. Genethon recently launched a gene therapy trial for this disease.

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Telethon 2022: for Lucie, “a real opportunity, thanks to advances in research”

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Publication: Genethon helps identify two vectors optimized for muscles via a European H2020 program

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First Patient Dosed in Phase 1/2 Clinical Trial in Europe of Gene Therapy for LGMD-R9

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The Genethon platforms obtain ISO 9001 certification

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Anne Galy receives the WAS Foundation Callahan Award for her work on Wiskott-Aldrich syndrome

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Sickle-cell anemia: a project involving the Gene editing team has just secured European funding

The “EDITSCD” project, that aims to improve gene editing strategies for sickle-cell anemia, this hereditary disease of the red blood cells, has just been selected by the European Union. It will involve a dozen or so research laboratories in Europe, among then the Gene editing team (Inserm UMR 951) headed by Mario… (read more)

Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain

To mark International Rare Disease Day, Genethon identifies the most urgent priorities in overcoming these obstacles.

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Five post-doctoral positions open at Genethon, a leader in gene therapy for rare diseases

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From 2021, a seminal year for Genethon, to 2022, a year of challenges

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Myotubular myopathy: “Today my son Jules is able to stand”

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Pierre, suffering from limb girdle muscular dystrophy: “For me, a cure would mean no more losing”

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Spinal muscular atrophy: for Victoire’s mom, treatment was a magical moment

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