This content is machine translated Spinal muscular atrophy The disease in 2022 in the light of new therapeutic options In spinal muscular atrophy, insufficient SMN protein is provided due to a defect or loss of the SMN1 gene. As a result, motor neurons in the spinal cord and lower…
View Post 2 min This content is machine translated Spinal muscular atrophy Single-agent gene therapy shows transformative therapeutic benefit New trial data demonstrate age-appropriate development and achievement of motor milestones in presymptomatic children with SMA using single-agent gene therapy, without any respiratory or nutritional support or serious treatment-related adverse…
View Post 3 min This content is machine translated Spinal muscular atrophy Clinical experience speaks for individual therapy management Spinal muscular atrophy is a rare genetic disorder characterized by the loss of motor neurons in the spinal cord and lower brainstem. In the meantime, experts agree that therapy management must…
View Post 3 min This content is machine translated Spinal muscular atrophy Therapy goals in adult SMA patients firmly in view Spinal muscular atrophy, although a rare condition, is among the most common genetic causes of death in infants and young children. However, adolescents and adults can also be affected by…
View Post 4 min This content is machine translated Orphan disease Excellent and innovative treatment for spinal muscular atrophy Spinal muscular atrophy is associated with rapid loss of motor neurons, resulting in, among other things, impaired breathing. It represents the most common genetic cause of infant and young child…
View Post 2 min This content is machine translated Spinal muscular atrophy SMA in adults: the road to personalized treatment Adults with spinal muscular atrophy form a clinically and genetically very heterogeneous group. A common feature is the progressive loss of motor anterior horn cells in the spinal cord, resulting…
View Post 3 min This content is machine translated Spinal muscular atrophy Promising therapeutic approach in the pipeline New results from the FIREFISH and SUNFISH studies suggest that there is hope for children with spinal muscular atrophy.
