A new study by US researchers has found a new novel drug that shows hope for infants suffering from rare, fatal muscle disease. This new experimental drug may have the potential to improve the quality of life for infants suffering from a rare, lethal neuromuscular disorder. Spinal muscular atrophy is a genetic disease that affects around one in every 11,000 births. It affects the nerve cells in the spinal cord that connect to the muscles, causing them to waste away resulting in progressive muscle weakness and difficulty in breathing and eating.
Infantile-onset, which is the most severe form of the disease, occurs from a defect in the gene called SMN2, responsible for producing survival motor neurone (SMN) -- a protein critical for normal cell function. It affects babies under the age of six months. Less than a quarter of those diagnosed with the disease will live up to two years without…
