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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.
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.
Illustration for article: Telethon 2012 : New drug trials in 2013

Telethon 2012 : New drug trials in 2013

Young patients with immunodeficiencies, rare diseases of the blood and brain, have already benefited from innovative therapies stemming from research driven by the Telethon. Thirty-six trials are in progress or in preparation for nearly 30 diseases.
Illustration for article: AFM-Telethon to be the first non-profit stakeholder in the world of medicine

AFM-Telethon to be the first non-profit stakeholder in the world of medicine

A few days before the launch of the Telethon on December 7th and 8th , the AFM-Telethon organised a press conference in Paris, presenting the Association’s challenges for 2013 and the researchers that it supports.
Illustration for article: The AFM-Telethon has been rewarded for its transparent and responsible communication

The AFM-Telethon has been rewarded for its transparent and responsible communication

At the ceremony of the 26th Communication and Entreprise awards 2012, the AFM-Telethon received the responsible communication award and a distinction for its 2011 Annual Report.
Illustration for article: Shinya Yamanaka, 2012 Nobel Prize in Medicine visits the I-Stem laboratory

Shinya Yamanaka, 2012 Nobel Prize in Medicine visits the I-Stem laboratory

On Friday November 9th, the Japanese physician and researcher, Shinya Yamanaka, who was awarded the Nobel Prize in Medicine on October 8th for his work on IPS cells (Induced Pluripotent Stem cells), visited the I-Stem laboratory in Evry.
Illustration for article: Decree “advanced therapy medicinal products”: a new milestone for the AFM-Telethon and laboratory Genethon!

Decree “advanced therapy medicinal products”: a new milestone for the AFM-Telethon and laboratory Genethon!

With this decree, Genethon will be able to accelerate the production of gene therapy for human trials through its center, Genethon Bioprod, which has the largest capacity in the world in this field.
Illustration for article: Généthon receives the 2012 French Galien Prize

Généthon receives the 2012 French Galien Prize

Généthon receives the 2012 French Galien Prize for its innovative treatments of rare diseases using gene therapy and awarded it the prize in the category of “Medicines destined for rare diseases – cell therapy and gene therapy.”
Illustration for article: The Biotherapies Institute, a unique and exceptional task force

The Biotherapies Institute, a unique and exceptional task force

Launched in 2012, the Biotherapies Institute gathers the expertise of existing laboratories to accelerate and manage the development of innovative treatments for rare diseases.
Illustration for article: Call for proposals ends on Septembre 28th

Call for proposals ends on Septembre 28th

AFM-Telethon will support research projects from both French and foreign groups. Proposals aim to support fundamental research on neuromuscular system, develop therapeutic approach and improve care management of patient.