Advances in myotonic dystrophy type 1 – June 2024

This disease, which is also known as Steinert disease, mainly affects the muscles but can also impact other organs to a greater or lesser extent. DM1 is a “multisystem” disorder, meaning it affects several different organs (muscles, the heart, the respiratory system, the digestive system, the endocrine system and the nervous system). Muscles decrease in volume, become weak (dystrophy) and have difficulty relaxing after they contract (myotonia).

Symptoms vary greatly from patient to patient, with some forms remaining asymptomatic (no symptoms) until old age while others are very severe and start at birth.

This document, published to coincide with the AFM-Téléthon General Meeting 2024, presents myotonic dystrophy type 2 research news from the past year (ongoing studies and clinical trials, scientific and medical publications, etc.).

 

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