What Is Duchenne Muscular Dystrophy? Muscular dystrophies are a group of diseases that make muscles weaker and less flexible over time. Duchenne muscular dystrophy (DMD) is the most common type.
Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...
This follows confirmation that no appeals were received against the Final Draft Guidance (FDG) recommendation announced on December 10, 2024. Following this, Santhera has already started launch ...