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The 30th of September is Limb-Girdle Muscular Dystrophy Awareness Day. At this occasion, the LGMD Interest Group of the AFM-Téléthon is planning an information day about these diseases in Evry. 

The 10th World Duchenne Awareness Day, on September 7, is a call to action to break down the physical, social and healthcare barriers faced by the 250,000 people around the world affected by this disease. It's also an opportunity to take stock of advances in research into this disease, which is emblematic of our Association. 

In an article published in July 2023, an Italian researcher traces the birth and development of myology. This medical science of muscle and its diseases has developed over time, not only thanks to the advent of tools and technologies that improve our knowledge of muscle, but also thanks to the international collaborations that have marked its history.

ILTOO Pharma and the European consortium MIROCALS have just announced the signing of a license agreement for the development of a low-dose Interleukin 2 as a potential treatment for amyotrophic lateral sclerosis (ALS). The AFM-Telethon helped finance this clinical study, which shows an improvement in patient survival. 

It is a positive commitment, but France must set an example by perpetuating and amplifying a truly ambitious national policy.

On 16 December 2021, the UN adopted the first-ever UN Resolution on “Addressing the Challenges of Persons Living with a Rare Disease and their Families.” A huge recognition for families with rare diseases.

On February 28, International Rare Disease Day will put the spotlight on the rare diseases community throughout the world. At this occasion, AFM-Telethon revisits thirty years of pioneering research and innovation carried out by its laboratory, Genethon, which has developed a high-level expertise in researching and developing preclinical and clinical gene therapy treatments for rare diseases.

Enabling early detection and diagnosis of the disease is the aim of the new European Alliance for Newborn Screening in Spinal Muscular Atrophy (SMA NBS Alliance), launched on August 31st by SMA Europe, of which AFM-Telethon is a member.

Four types of myositis that consider all the clinical criteria of patients have been defined. A new classification that paves the way for reliable diagnosis and personalised treatments for patients.