News

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

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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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Preliminary results of clinical trial for rare liver disease Crigler-Najjar syndrome presented at ESGCT congress

After a first presentation at the International Liver Congress last June, new preliminary results have just been presented at the European Society of Geneand Cell Therapy (ESGCT) congress by Dr d’Antiga, one of the investigators of the trial (Bergamo, Italy). Based on initial observations, the drug candidate is well tolerated and the first… (read more)

Cholesterol Metabolism: A Potential Therapeutic Target in Duchenne Muscular Dystrophy

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Genethon’s teams present their progress at the ASGCT meeting

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Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT 0004 for Duchenne Muscular Dystrophy

Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT 0004 for Duchenne Muscular Dystrophy

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