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Illustration for article: Myositis: A new classification system based on phenotypic, biological and immunological criteria

Myositis: A new classification system based on phenotypic, biological and immunological criteria

Illustration for article: Overview of Myology 2019

Overview of Myology 2019

Myology2019, the sixth edition of the international congress of myology organized by AFM-Telethon, took place in Bordeaux, France, from March 25 to 28.
Illustration for article: 85 844 117 euros - #Telethon2018 #Thankyou #Amazingdonors

85 844 117 euros - #Telethon2018 #Thankyou #Amazingdonors

The final result of the Telethon 2018 reaches 85 844 117 euros*. This extraordinary result, achieved in particularly difficult circumstances, demonstrates once again the utmost loyalty of the French people towards the struggle of affected families.
Illustration for article: Sixth Congress of Myology

Sixth Congress of Myology

From March 25 to 28, AFM-Téléthon is organizing Myology 2019, a congress dedicated to muscles, and gathering experts from around the world.
Illustration for article: AFM-Téléthon honored as “Pioneer in Technology Development”

AFM-Téléthon honored as “Pioneer in Technology Development”

Los Angeles, March, 16, 2019 - AFM-Téléthon received the « Pioneer in Technology Development » award for its role in the development of innovative therapies for neuromuscular and rare diseases at the Annual World Congress of Society for Brain Mapping and Therapeutics (SBMT-Brain and Spine).
Illustration for article: Treatment of X-CGD : positive results of the gene therapy initiated by Genethon

Treatment of X-CGD : positive results of the gene therapy initiated by Genethon

Genethon, AFM-Telethon’s laboratory, at the origin of the gene therapy for this rare immunodeficiency disorder, welcomes the very encouraging results announced by Orchard Therapeutics, a biopharmaceutical company.
Illustration for article: Myology 2019

Myology 2019

The 6th edition of the international congress of myology, from 25 to 28 March at the Palais des Congrès of Bordeaux-Lac.
Illustration for article: The 2018 Telethon counter reaches €69 290 089.

The 2018 Telethon counter reaches €69 290 089.

The year where the Telethon shares its greatest victories, a very difficult social context weakens its counter of donations.
Illustration for article: Co-administration of AAV Vectors with SVP-Rapamycin enables vector re-administration in pre-clinical gene therapy

Co-administration of AAV Vectors with SVP-Rapamycin enables vector re-administration in pre-clinical gene therapy

Généthon, founded by the AFM-Téléthon, and Selecta Biosciences, a clinical-stage biopharmaceutical company, today announced that Nature Communications has published their jointly authored paper entitled “Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration”. A world first !
Illustration for article: The Telethon 2018 focuses on victories

The Telethon 2018 focuses on victories

In 2018, the Telethon celebrates advances in research and highlights civic, scientific and therapeutic Victories. Join us on December 7 and 8, 2018.
Illustration for article: The “Cure Through Innovation 2018” booklet: all AFM-Téléthon at a glance

The “Cure Through Innovation 2018” booklet: all AFM-Téléthon at a glance

The Cure Through Innovation 2018 booklet is now on line.
Illustration for article: AFM is turning 60 this year

AFM is turning 60 this year

Sixty years ago to this day, the creation of the AFM revolutionized the destiny of rare diseases
Illustration for article: The 31th Telethon edition reaches a final collection of 89,189,384 euros

The 31th Telethon edition reaches a final collection of 89,189,384 euros

On 8th and 9th December 2017, the Telethon enthralled the public throughout France.
Illustration for article:  Spinal muscular atrophy : AveXis enters into licensing agreement with Genethon

Spinal muscular atrophy : AveXis enters into licensing agreement with Genethon

AveXis, Inc. and Genethon today announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy delivery of AAV9 vector into the central nervous system (CNS) for the treatment of spinal muscular atrophy (SMA).
Illustration for article: Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome

Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome

Généthon, a laboratory created by AFM-Téléthon, starts a European phase I/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease.
Illustration for article: Myotubular Myopathy

Myotubular Myopathy

Genethon welcomes interim data of a phase I / II clinical trial with a gene therapy product developed in its laboratories
Illustration for article: Cure through innovation 2017 is now available online

Cure through innovation 2017 is now available online

Every year, the “Cure through innovation” booklet synthetically presents the Association, recalling particularly the key points of the annual report.
Illustration for article:  International Scientific Congress on spinal muscular atrophy, from 25 to 27 January 2018,  in Krakow.

International Scientific Congress on spinal muscular atrophy, from 25 to 27 January 2018, in Krakow.

SMA Europe welcome you to Krakow for International Scientific Congress on spinal muscular atrophy, which will take place from 25 to 27 January 2018 in Auditorium Maximum of the Jagiellonian University.
Illustration for article: Gene therapy: Microdystrophin restores muscle strength in Duchenne muscular dystrophy

Gene therapy: Microdystrophin restores muscle strength in Duchenne muscular dystrophy

Illustration for article: SMA: European marketing authorisation for SpinrazaTM (nusinersen)

SMA: European marketing authorisation for SpinrazaTM (nusinersen)

SpinrazaTM (nusinersen), the first and only treatment for spinal muscular atrophy (SMA), is now available in Europe.