Apparent at birth or in the first few months of life, congenital muscular dystrophies (CMD) are a group of very diverse, rare diseases characterised by progressive muscle weakness in the trunk and limbs. They may also affect other organs such as the heart, brain and eyes.
Common symptoms are muscle weakness with hypotonia causing impaired motor skills and often associated with breathing and/or feeding problems, and orthopaedic deformities such as contractures causing skeletal deformities. These symptoms vary from one type of CMD to another and from patient to patient, even within the same family.
This document presents congenital muscular dystrophy research news from the past year (ongoing observational studies and clinical trials, scientific and medical publications, etc.).
Access the document Advances in congenital muscular dystrophies – June 2024
