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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: 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.
Illustration for article: Telethon fundraising event 2015 : 80 251 183 euros. More than ever THANK YOU!

Telethon fundraising event 2015 : 80 251 183 euros. More than ever THANK YOU!

At the end of a 30-hour TV marathon, the Telethon counter displays an extraordinary result of 80 251 183 euros.
Illustration for article: INGESTEM international congress and cell therapie

INGESTEM international congress and cell therapie

INGESTEM, I-Stem and biotherapies: 1st congress november 19-20th, 2015
Illustration for article: Cell therapy in France & I-Stem: just 10 years from research to first clinical trials

Cell therapy in France & I-Stem: just 10 years from research to first clinical trials

Illustration for article: Ryr-1 Foundation, a very active patient group

Ryr-1 Foundation, a very active patient group

This American foundation initiated a patient self-reporting registry and is well-known to support research and promote clinical trials of potential therapies.
Illustration for article: A new step forward to gene-medicine for AFM-Telethon : to produce and cure

A new step forward to gene-medicine for AFM-Telethon : to produce and cure

AFM-Telethon has decided to take up a new challenge: to produce gene-medicines from innovative biotherapies developed in the laboratories part of its Biotherapies Institute for Rare Diseases at an industrial scale, and to give patients suffering from rare genetic diseases access to them at a fair and contained price. With this in view, the association will co-fund with Bpifrance the creation of the largest European center for the development and manufacturing of gene and cell therapies. This ambitious project opens up a new key stage on the way to the gene-medicine for the organization.