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”

Lucie has spinal muscular atrophy. A year ago, she was able to benefit from a gene therapy treatment based on technologies developed at Genethon. Lucie and her parents will be ambassadors of the 2022 Telethon.

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

Making gene therapy vectors more specific is a major challenge in gene therapy. Edith Renaud-Gabardos, under the direction of Ana Buj-Bello, an Inserm Research Director, and several other Genethon collaborators, co-authored a publication in Science Advances demonstrating the optimized effectiveness of two capsids for muscle… (read more)

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

Study to treat FKRP-related limb-girdle muscular dystrophy type 2I/R9 represents 30 years of research and is being conducted by Genethon spinout Atamyo Therapeutics.

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The Genethon platforms obtain ISO 9001 certification

As of July 8, 2022, the Imaging-Cytometry, Preclinical Evaluation and Technological Development platforms have been officially ISO 9001 certified. An additional guarantee of the quality of the activities of these platforms.

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

The WAS Foundation awarded the Callahan Prize for outstanding service to Anne Galy, head of the Immunology and biotherapies team. She has worked for more than 20 years on Wiskott-Aldrich syndrome, a severe immune deficiency of genetic origin.

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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 Amendola at… (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

A pioneer and leader in gene therapy for rare disease, Genethon, a non-profit organization, recruits 5 post-doctoral fellow to develop research projects the Inserm-Genethon-UEVE Integrare unit.

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

After a year of significant breakthroughs in 2021, 2022 sees Genethon continuing its efforts in face of the medical and technological challenges of gene therapy for rare diseases, helping patients and their families.

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

Suffering from myotubular myopathy, a disease that’s often fatal before the age of 2, Jules received a gene therapy treatment produced by research from Genethon, as part of a clinical trial in January 2020. His progress is spectacular.

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

Pierre is a 14-year-old middle-schooler. Four years ago he received the diagnosis on FKRP limb girdle muscular dystrophy. This progressive disease saps his strength little by little. At Genethon, Isabelle Richard’s team has worked for over 25 years on limb girdle muscular dystrophy. A first gene therapy treatment for Pierre’s disease could… (read more)

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

Suffering from spinal muscular atrophy, Victoire received a gene therapy treatment derived from technologies developed at Genethon. Her mom recounts the anguish of the diagnosis, the immense relief of the treatment and the little girl’s spectacular progress in the weeks following the injection.

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