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.
With the theme "Duchenne: breaking barriers", the day dedicated to raising awareness of Duchenne muscular dystrophy aims to encourage an open society, offering equal opportunities for all.
On September 7, the World Duchenne Organization will launch a WDAD documentary that portrays the lives of people living with Duchenne Muscular Dystrophy across the globe and how they are each breaking barriers in their personal lives.
To find out more about the Day, visit the dedicated website.
100 to 150 newborns affected by the disease in France every year
Duchenne muscular dystrophy is the most common neuromuscular disease. It begins in childhood and evolves very gradually: often, around the age of 3 or 4, the first muscles difficulties appear, sometimes with problems walking and increased tiredness. Over the years, the disease that causes muscles to become weaker until it affects the whole body.
Duchenne muscular dystrophy affects 4.78 people per 100,000. This means that between 100 and 150 newborn boys in France are affected by Duchenne muscular dystrophy every year.
Duchenne muscular dystrophy at the centre of innovative treatments
For this disease, the symbol of the AFM-Téléthon's fight, research is on progress and clinical trials are multiplying! Innovative treatments - gene therapy, gene surgery, oligonucleotides, etc. - are raising hopes of halting the progression of the disease.
After years of research, Généthon, the AFM-Téléthon laboratory, in collaboration with the gene therapy laboratory in Nantes and Royal Holloway in London, have developed a highly innovative gene therapy treatment to restore muscle strength in patients.
It involves the intravenous injection of the gene for micro-dystrophin (a shortened version of dystrophin) to restore the production of this protein, the absence of which leads to muscle degeneration. Trials are underway in France and the UK.
To find out more about Advances in Duchenne and Becker muscular dystrophy, click here.
Duchenne Muscular Dystrophy and the AFM-Téléthon
Duchenne muscular dystrophy is a disease that symbolises the fight of the French Muscular Dystrophy Association (AFM-Telethon), an association of activists, patients and parents of patients, affected by rare, progressive and significantly disabling genetic diseases, known as genetic neuromuscular diseases. Yolaine de Kepper, a mother of seven children, four of whom suffered from Duchenne muscular dystrophy (DMD), was the Association’s founder and first Chair.
Since its creation in 1958, the association has been fighting for a cure for rare diseases that have long been ignored and neglected by public authorities.
The discovery of the gene responsible for Duchenne muscular dystrophy in 1986 has been a crucial moment for AFM-Telethon, which then decided that genetic research would be the spearhead of its commitment to finding a cure and created the first Telethon in 1987 in order to have the financial resources to fund innovative and powerful research.
To know more about the history of AFM-Téléthon, read the page Our history