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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: 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.
Illustration for article: The 14th Société Française de Myologie days will take place in Bordeaux from November 23rd to 25th 2016

The 14th Société Française de Myologie days will take place in Bordeaux from November 23rd to 25th 2016

The Société Française de Myologie invites all myology specialists on November 23rd, 24th and 25th in Bordeaux.
Illustration for article: The final result of Telethon 2015 came up to €93,850,778!

The final result of Telethon 2015 came up to €93,850,778!

An outstanding result for this 29th edition
Illustration for article: Dr. Odile Boespflug-Tanguy, new president of the AFM-Téléthon Scientific Council

Dr. Odile Boespflug-Tanguy, new president of the AFM-Téléthon Scientific Council

The handover is occurring today at Myology 2016.
Illustration for article: Follow all the news from Myology 2016 !

Follow all the news from Myology 2016 !

Throughout the week, follow the news from Myology 2016 on this page !
Illustration for article: Myology 2016 starts today in Lyon!

Myology 2016 starts today in Lyon!

Follow all the news on Myology 2016 on this page!
Illustration for article: Myology 2016, 5th edition from March 14 to March 18 in Lyon

Myology 2016, 5th edition from March 14 to March 18 in Lyon

The 5th edition of the International Congress of Myology organised by AFM-Téléthon is taking place at the Palais des Congrès in Lyon, from March 14 to March 18 with the participation of over 800 international experts.
Illustration for article: Call for a Third Rare Diseases National Plan.

Call for a Third Rare Diseases National Plan.

While the press event for the 9th International Day of Rare Diseases, members of the Platform for Rare Diseases have called for a 3rd National Plan.
Illustration for article: Rare Disease Day 2016

Rare Disease Day 2016

On 29 February, patients around the world will celebrate the ninth annual Rare Disease Day. People living with a rare disease and their families, patient organisations, politicians, carers, medical professionals, researchers and industry come together in solidarity to raise awareness of rare diseases.