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Illustration for article: Save the date! MYOLOGY 2016 - Fifth International Congress of Myology

Save the date! MYOLOGY 2016 - Fifth International Congress of Myology

After Lille in 2011, AFM-Telethon is organizing its Fifth International Congress of Myology in Lyon, from 14th till 18th March 2016
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: 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).
Illustration for article: Telethon 2014 : 92 920 108 €

Telethon 2014 : 92 920 108 €

An exceptional result, a fundraiser success, audience and mobilization
Illustration for article: Sarepta Therapeutics Announces First Patient Dosed in Duchenne Muscular Dystrophy Patients

Sarepta Therapeutics Announces First Patient Dosed in Duchenne Muscular Dystrophy Patients

Sarepta Therapeutics Inc. a developer of RNA-­‐based therapeutics, announced that it has initiated dosing of SRP-­‐4053 in its first human trial, a Phase I/II study in Duchenne muscula rdystrophy (DMD).
Illustration for article: Effectiveness of innovative gene therapy treatment demonstrated in canine model of Duchenne muscular dystrophy

Effectiveness of innovative gene therapy treatment demonstrated in canine model of Duchenne muscular dystrophy

A collaboration involving three laboratories supported by the AFM-Telethon, Atlantic Gene Therapies (AFM-Telethon, Inserm UMR 1089, Université de Nantes,Nantes University Hospital), Généthon (Evry) and the Institute of Myology (Paris), demonstrated the effectiveness of an innovative gene therapy treatment in the canine model of Duchenne muscular dystrophy.
Illustration for article: Discover Telethon 2014 exceptional program

Discover Telethon 2014 exceptional program

An exceptional sponsor, 4 families, 30 hours of broadcast on France Télévisions and 2,000 volunteers in the starting blocks: Telethon 2014 promises a lot of surprises!
Illustration for article: Cure through innovation : our new version is online

Cure through innovation : our new version is online

Since it was created, AFM-Téléthon does all it can to accomplish its goals and its conviction that a cure is possible.Discover our new version of the brochure "Cure through innovation"
Illustration for article: International Call for Spinal Muscular Atrophy (SMA) Research Projects 2014

International Call for Spinal Muscular Atrophy (SMA) Research Projects 2014

This new Call will be open to any research project aimed at finding a therapy for Spinal Muscular Atrophy (SMA) or at elucidating the basic pathophysiological processes of the disease.
Illustration for article: European Medicines Agency recommends first-in-class medicine for treatment of Duchenne muscular dystrophy

European Medicines Agency recommends first-in-class medicine for treatment of Duchenne muscular dystrophy

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting a conditional marketing authorisation for Translarna (ataluren) an orphan-designated medicine for the treatment of Duchenne muscular dystrophy caused by nonsense mutations.