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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: 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.
Illustration for article:  New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs

New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs

A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a large animal model of the disease. This work, published today in Molecular Therapy, has been achieved thanks to donations from the French Telethon and the support of the Myotubular Trust.
Illustration for article: "Téléthon, et après"

"Téléthon, et après"

A brand new section dedicated to all the questions you may have about AFM-Telethon
Illustration for article: The 30th Telethon edition reaches a final collection of 92,740,769 euros

The 30th Telethon edition reaches a final collection of 92,740,769 euros

On 2nd and 3rd December 2016, the Telethon enthralled the public throughout France. The final collection figure now allows us to pursue the objective we set ourselves 30 years ago : to find a cure !
Illustration for article: YposKesi welcomes Alain Lamproye, an expert in industrial bioproduction, as its Chief Executive Officer

YposKesi welcomes Alain Lamproye, an expert in industrial bioproduction, as its Chief Executive Officer

YposKesi, the first French pharmaceutical company to develop and manufacture gene and cell therapy products for rare diseases, created by the nonprofit organization AFM-Téléthon and the SPI fund managed by Bpifrance, have announced the appointment of Alain Lamproye as its Chief Executive Officer.
Illustration for article: Prize4Life Announces ALS Treatment Prize Winner

Prize4Life Announces ALS Treatment Prize Winner

Prize4Life, a nonprofit organization whose mission is to accelerate the discovery of treatments and a cure for ALS (Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease), today announced the winner of its $1M Avi Kremer ALS Treatment Prize. The winners are the team of Drs. Martine Barkats and Maria-Grazia Biferi, from the Institute of Myology U974 INSERM UMPC in Paris, France.
Illustration for article: The 2017 Rare Disease Day Video

The 2017 Rare Disease Day Video

Illustration for article: Spinraza drug authorization: Patients’ organization AFM-Telethon welcomes a major step but calls upon full transparency by Biogen regarding the price claimed

Spinraza drug authorization: Patients’ organization AFM-Telethon welcomes a major step but calls upon full transparency by Biogen regarding the price claimed