A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study published in The Lancet Neurology. Spinal muscular atrophy (SMA) is a rare genetic disorder that affects motor neurons – nerve cells in the spinal cord responsible for controlling voluntary…
New research from the Exercise Medicine Research Institute at Edith Cowan University (ECU) has highlighted that children undergoing cancer treatment often lose skeletal muscle at a time when they should be rapidly gaining it, a loss that could increase treatment complications and raise the risk of long-term health problems. Both the cancer itself and…
Texas Children’s is pleased to announce that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency. Aromatic l-amino acid decarboxylase (AADC) deficiency is an extremely rare, inherited neurological disorder that prevents the brain from producing dopamine and serotonin – essential chemicals for controlling movement, mood and basic…
Spinal muscular atrophy (SMA) is a rare genetic condition that causes progressive muscle weakness, which, when untreated, prevents infants with the most severe form from gaining motor development – never gaining the ability to sit – and typically leads to death before 2 years of age. The oral drug risdiplam benefits symptomatic patients with improved…