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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: Families and researchers applaud French people

Families and researchers applaud French people

An impressive result that embodies thirty hours of meetings and amazing celebrations in 10,000 cities and at the foot of the Eiffel Tower, in a magical bubble.
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: 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.
Illustration for article: Telethon 2015: 4 families, 4 stories, 4 fights against rare disease

Telethon 2015: 4 families, 4 stories, 4 fights against rare disease

Four families, all affected by rare diseases, will be the ambassadors of thousands, all gathered around the message: “The Fight of Parents, The Life of Children.”
Illustration for article: World premiere : differentiation of pluripotent stem cells to muscle fiber

World premiere : differentiation of pluripotent stem cells to muscle fiber

This major breakthrough in neuromuscular diseases has just been published August 3, 2015 in Nature Biotechnology.
Illustration for article: A new trial in gene therapy led by UCLA in partnership with Genethon

A new trial in gene therapy led by UCLA in partnership with Genethon

10 children with an immunodeficiency disorder, commonly referred to as X-linked CGD, will be enrolled to receive a stem cell gene therapy, due to Dr Kohn's team and Genethon.
Illustration for article: ASGCT 18 th Annual Meeting 2015: Ana Buj Bello receives the Outstanding New Investigator Award

ASGCT 18 th Annual Meeting 2015: Ana Buj Bello receives the Outstanding New Investigator Award

This award recognizes four researchers every year for the quality and importance of their work in gene and cell therapy.
Illustration for article: A breakthrough in gene therapy : Crigler-Najjar syndrome

A breakthrough in gene therapy : Crigler-Najjar syndrome

Federico Mingozzi, head of the Immunology and Liver Gene Therapy team at Généthon, the laboratory created by the AFM-Téléthon, presented work done in collaboration with an Italian and Dutch teams showing long-term correction of a genetic defect causing toxic buildup of bilirubin in murine and rat models of Crigler-Najjar syndrome.
Illustration for article: New gene therapy success in a rare disease

New gene therapy success in a rare disease

French teams from CIC Biothérapie (AP-HP/Inserm), from pediatric hematology department of Necker Hospital for Children (AP-HP), led by Marina Cavazzana, Salima Hacein Bey Albina and Alain Fischer and from Genethon led by Anne Galy (Genethon/Inserm UMR-S951), and English teams from UCL Institute of Child Health and Great Ormond Street Hospital in London led by Adrian Thrasher and Bobby Gaspar demonstrated the efficacy of gene therapy treatment for Wiskott-Aldrich Syndrome (WAS).