Question 37023 — diseases

Summary

Ms. Huguette Tiegna questions the Minister of Solidarity and Health about Duchenne muscular dystrophies, particularly called Duchenne myopathy. On the occasion of World Rare Disease Day, February 28, 2021, it is important to talk about this disease. Duchenne muscular dystrophy is a form of progressive muscular dystrophy that becomes more widespread over the years. This hereditary disease, with recessive transmission linked to X chromosome, is transmitted only to boys. Thus, in France, each year 150 newborns present this disease and affects 2,500 people, it is the most widespread myopathy in children. This disease, often unknown, usually manifests itself from the age of three in young boys and presents various symptoms such as muscle atrophy, progressive muscular weakness of the limbs and trunk, then damage to the respiratory and cardiac muscles. Thus, until now, the treatments offered were palliative, in particular rehabilitation (massage, physiotherapy), additional surgery, equipment, ventilation to facilitate the flexibility of the rib cage or even nutritional treatments in order to adapt the diet and the way of swallowing and eating. However, several clinical trials are underway, in particular the gene therapy at the origin of Généthon, the AFM-téléthon laboratory, dedicated to the development and design of these products. In November 2020, they received authorization from the National Agency for the Safety of Medicines and Health Products (ANSM) to start, in France, a clinical trial on Duchenne muscular dystrophy. Around a hundred young people will be followed over a period of three months to three years. As such, she would like to know the recruitment methods to join the clinical trial and what the State's share of funding will be given the cost of this experimental treatment for families, who already have to face significant expenses in the context of this disease.

Machine translation from French. The official text remains authoritative.

Timeline

No timeline events have been ingested for this record yet.

Votes

No vote records are attached yet.

Versions

No version snapshots stored. Document URLs remain at the source.

Documents

No documents linked.

Sponsors

No sponsors or actors listed by the source.

Related records

No cross-record relationships stored yet.