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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: 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: 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.”
Illustration for article: International Call for Spinal Muscular Atrophy (SMA) Research Projects 2013

International Call for Spinal Muscular Atrophy (SMA) Research Projects 2013

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: Généthon becomes the first not-for-profit to obtain authorization to be a pharmaceutical manufacturer

Généthon becomes the first not-for-profit to obtain authorization to be a pharmaceutical manufacturer

The AFM-Telethon laboratory, has received the authorization delivered by the National Agency for Drug Safety (ANSM) to become a pharmaceutical manufacturer.
Illustration for article: Myotubular Trust is supporting Généthon in developing gene therapy for myotubular myopathy

Myotubular Trust is supporting Généthon in developing gene therapy for myotubular myopathy

The European charity Myotubular Trust announces its support to the development of a pre-clinical gene therapy trial for myotubular myopathy.
Illustration for article: Biotherapies and rare diseases: focus on a medical revolution

Biotherapies and rare diseases: focus on a medical revolution

For more than 20 years, the AFM-Telethon supports the development of innovative therapies that benefit rare diseases and medicine as a whole.
Illustration for article: Seed capital fund: an incentive to treat rare diseases

Seed capital fund: an incentive to treat rare diseases

The AFM-Telethon and FNA, a fund managed by CDC Entreprises, a unit of the Public investment bank, have launched the first seed capital fund dedicated to innovative biotherapies and rare diseases.
Illustration for article: Drisapersen: improvement in walking distance in patients with Duchenne muscular dystrophy

Drisapersen: improvement in walking distance in patients with Duchenne muscular dystrophy

GSK has announced a significant improvement in walking distance in patients with Duchenne muscular dystrophy treated for one year with drisapersen, an orphan drug that promotes exon skipping. The Institute of Myology collaborated in this phase II trial.
Illustration for article: Thanks to your donations, new actions are being launched in 2013

Thanks to your donations, new actions are being launched in 2013

With its Genethon laboratory, the AFM-Telethon is taking up the challenge of becoming the first patient association to produce gene therapy medicines. Three new trials will also be added to those already in progress.
Illustration for article: Telethon 2012: 88,156,400 euros donated

Telethon 2012: 88,156,400 euros donated

The final amount donated largely exceeds the pledges recorded at the end of the broadcast. A remarkable result as the AFM-Telethon prepares to meet new challenges for 2013.