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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: 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.
Illustration for article: Genosafe celebrates its 10th anniversary

Genosafe celebrates its 10th anniversary

Founded in 2004 by GENETHON and AFM-Telethon, Genosafe is a service company whose mission is to advise and assist its clients at all stages of drug development innovating therapy products.
Illustration for article: 8-10 may 2014 : ECRD 2014 : The European Conference on Rare Diseases & Orphan Products

8-10 may 2014 : ECRD 2014 : The European Conference on Rare Diseases & Orphan Products

The ECRD is the unique platform reaching across all rare diseases and across all European countries, to bring together all stakeholders – patients’ representatives, academics, health care professionals, industry, payers, regulators and policy makers.
Illustration for article: Trophos will present results of pivotal phase II/III study of olesoxime in spinal muscular atrophy patients at the American Academy of Neurology (AAN)

Trophos will present results of pivotal phase II/III study of olesoxime in spinal muscular atrophy patients at the American Academy of Neurology (AAN)

Illustration for article: Efficacy of gene therapy demonstrated in canine and murine models of myotubular myopathy

Efficacy of gene therapy demonstrated in canine and murine models of myotubular myopathy

Illustration for article: Téléthon 2013 : 78 341 598 euros

Téléthon 2013 : 78 341 598 euros

Thank you ! A huge thank you to all the volunteers who have joined forces everywhere in France, all the donors whose supports and give us fantastic and numerous expressions of encouragement during all those 30 hours.
Illustration for article:  A new clinical trial lauched based on Telethon donations

A new clinical trial lauched based on Telethon donations

A phase gene therapy clinical trial has just been lauched for Sanfilippo B syndrome, a rare genetic brain disease.
Illustration for article: Discover Telethon 2013 program a few days before kick- off

Discover Telethon 2013 program a few days before kick- off

Telethon 2013 is foremost 5 families, 5 stories, 5 fightings. Families involved in the disease, ambassadresses of thousands of others,united around a same message: “the fight of the parents, life of the children”
Illustration for article: Telethon 2013: 5 families, 5 stories, 5 fights against rare disease

Telethon 2013: 5 families, 5 stories, 5 fights against rare disease

Five 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.”