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Illustration for article: Take part in Génération Téléthon

Take part in Génération Téléthon

Post your Génération Téléthon photo on the website of the 30th Téléthon.
Illustration for article: Here are the faces of Telethon 2016

Here are the faces of Telethon 2016

Leo, Mandine, Elena, Matthieu… they come from different horizons, with their one stories, but they all have one goal: fighting the disease which affects them. They are Generation Telethon.
Illustration for article:  New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs

New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs

A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a large animal model of the disease. This work, published today in Molecular Therapy, has been achieved thanks to donations from the French Telethon and the support of the Myotubular Trust.
Illustration for article: "Téléthon, et après"

"Téléthon, et après"

A brand new section dedicated to all the questions you may have about AFM-Telethon
Illustration for article: The 30th Telethon edition reaches a final collection of 92,740,769 euros

The 30th Telethon edition reaches a final collection of 92,740,769 euros

On 2nd and 3rd December 2016, the Telethon enthralled the public throughout France. The final collection figure now allows us to pursue the objective we set ourselves 30 years ago : to find a cure !
Illustration for article: YposKesi welcomes Alain Lamproye, an expert in industrial bioproduction, as its Chief Executive Officer

YposKesi welcomes Alain Lamproye, an expert in industrial bioproduction, as its Chief Executive Officer

YposKesi, the first French pharmaceutical company to develop and manufacture gene and cell therapy products for rare diseases, created by the nonprofit organization AFM-Téléthon and the SPI fund managed by Bpifrance, have announced the appointment of Alain Lamproye as its Chief Executive Officer.
Illustration for article: Prize4Life Announces ALS Treatment Prize Winner

Prize4Life Announces ALS Treatment Prize Winner

Prize4Life, a nonprofit organization whose mission is to accelerate the discovery of treatments and a cure for ALS (Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease), today announced the winner of its $1M Avi Kremer ALS Treatment Prize. The winners are the team of Drs. Martine Barkats and Maria-Grazia Biferi, from the Institute of Myology U974 INSERM UMPC in Paris, France.
Illustration for article: The 2017 Rare Disease Day Video

The 2017 Rare Disease Day Video

Illustration for article: Spinraza drug authorization: Patients’ organization AFM-Telethon welcomes a major step but calls upon full transparency by Biogen regarding the price claimed

Spinraza drug authorization: Patients’ organization AFM-Telethon welcomes a major step but calls upon full transparency by Biogen regarding the price claimed

Illustration for article: “This 30th Telethon launches a generation of finders!”

“This 30th Telethon launches a generation of finders!”

The 30th Telethon counter displayed €80,319,113 after the 30-hour TV program.
Illustration for article: Here we go for Telethon 2016

Here we go for Telethon 2016

The 30-hour-long Telethon show starts in less than an hour !
Illustration for article: YposKesi, the 1st French industrial pharmaceutical company for producing gene and cell therapy drugs for rare diseases

YposKesi, the 1st French industrial pharmaceutical company for producing gene and cell therapy drugs for rare diseases

AFM-Téléthon and the SPI fund, managed by Bpifrance under the Programme d’Investissement d’Avenir, are creating YposKesi, the first French industrial pharmaceutical company dedicated to producing gene and cell therapy drugs for rare diseases.
Illustration for article: Garou, mentor of Telethon 2016

Garou, mentor of Telethon 2016

For the 30th Telethon, Garou will be the ambassador of all previous mentors! Telethon 2016 will take place on December 2nd and 3rd, 2016. Save the date!
Illustration for article: The Myotubular Trust 7th call for projects

The Myotubular Trust 7th call for projects

The Myotubular Trust is launching a new call for projects. This call is open to international and innovative projects.
Illustration for article: 30th anniversary of the DMD gene discovery

30th anniversary of the DMD gene discovery

Thirty years ago today, a team of researchers announced the discovery of the gene involved in Duchenne muscular dystrophy. Throwback to the day that led genetics into a new era: the era of innovative therapies and the hope to one day cure this disease.
Illustration for article: September 7th : World Duchenne Awareness Day

September 7th : World Duchenne Awareness Day

Organised by UPPMD along with numerous associations, September 7th is the World Duchenne Awareness Day, an occasion to take part to the event and share your testimonies. Get involved and display the red balloon.
Illustration for article: Cure through innovation 2016 is now available online

Cure through innovation 2016 is now available online

The “Cure through innovation” booklet is now available online. It outlines the action carried out by the Association.
Illustration for article: Long term correction of hyperbilirubinemia in animal models of Crigler-Najjar syndrome after AAV vector-mediated liver gene transfer

Long term correction of hyperbilirubinemia in animal models of Crigler-Najjar syndrome after AAV vector-mediated liver gene transfer

Dr. Federico MINGOZZI, Team Leader of the Immunology and Liver Gene Transfer unit at Généthon, (Inserm U951/UPMC), France, has demonstrated the long-term efficacy of an optimized AAV-UGT1A1 vector for the correction of Crigler-Najjar syndrome (CN) in two different animal models of the disease. This work, published in July 20, 2016 in Molecular Therapy Methods and Clinical Development was supported by the AFM-Telethon.
Illustration for article: Innovative biotherapies: towards high-precision medicine

Innovative biotherapies: towards high-precision medicine

A team of researchers from the I-Stem laboratory has identified, thanks to the screening of a cellular neuronal model derived from stem cells, therapeutic compounds for an autistic disorder of genetic origins, opening the way towards high-precision medicine.
Illustration for article: First European market authorization for a gene therapy medication for a immunodeficiency

First European market authorization for a gene therapy medication for a immunodeficiency

Strimvelis, a gene therapy drug for a immunodeficiency, has been granted a European market authorization on May 27th, 2016.