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Illustration for article: Trophos announces top-line results of pivotal trial of olesoxime in spinal muscular atrophy

Trophos announces top-line results of pivotal trial of olesoxime in spinal muscular atrophy

Promising results for the maintenance of motor function in spinal muscular atrophy (SMA) -a rare, serious and debilitating pediatric-onset neurodegenerative disease
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: 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: 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: 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.
Illustration for article: Congenital adrenal hyperplasia: support for a new rare disease

Congenital adrenal hyperplasia: support for a new rare disease

The AFM-Telethon is providing financial support to the International Fund for research on Congenital Adrenal Hyperplasia (IFCAH), the association of parents of patients affected by this disease. The research projects could be useful for other rare diseases.
Illustration for article: International Rare Disease Day: See you on February 28th

International Rare Disease Day: See you on February 28th

A rare disease affects nearly 30 million people in Europe and 3 million in France. On February 28th, more than 60 countries will be mobilised around a single message: "Rare Disorders without Borders."
Illustration for article: The AFM-Telethon reinforces its Scientific Council

The AFM-Telethon reinforces its Scientific Council

There has been a 42% increase in new members of the 7th Scientific Council of the AFM-Telethon and additional international experts. An opening at the international level to accelerate the development of innovative treatments to cure the sick and keep commitments to donors.
Illustration for article: Rare diseases : 38 million Euro funding for research

Rare diseases : 38 million Euro funding for research

European Union has announced 38 million Euro funding for research towards new treatments and for the development of IRDiRC, the central global rare disease hub involving 70 institutions – including AFM-Telethon.
Illustration for article: Telethon 2012: 81,065,239€. Thank you for having met the challenge

Telethon 2012: 81,065,239€. Thank you for having met the challenge

This year’s Telethon will remain the challenge of all challenges: the challenge of donors and volunteers in the face of crisis, the challenge of researchers and families in the face of disease ... Together, we have tackled them, with determination and courage, all united by the same desire: win!
Illustration for article: Telethon 2012 : "Dare to win"

Telethon 2012 : "Dare to win"

“Dare to win” is the campaign slogan for the Telethon 2012. It is also the families’ cry of hope that will be launched to the French public on December 7th and 8th.