SMN1-related proximal spinal muscular atrophy is a type of proximal spinal muscular atrophy, a rare group of genetic diseases causing degeneration of the nerve cells conveying, from the spinal cord to the muscles, messages ordering movement: the peripheral motor neurons. A decrease in the number of muscle fibres, which cannot survive without innervation, causes a lack of strength and muscle wasting (muscular atrophy). There are four types of SMN1-related proximal spinal muscular atrophy, according to age of onset and severity of the signs.
This document presents research news from the past year regarding SMN1-related proximal spinal muscular atrophy: ongoing clinical trials or studies, clinical/scientific publications, international symposia, etc.
Access the document Advances in SMN1-related proximal spinal muscular atrophy
