Metabolic abnormalities, including the buildup of fat in the liver and other issues, occur before the loss of motor neurons,…
Articles by Katherine Poinsatte, PhD
Fertility issues and low sperm counts are common in men with spinal muscular atrophy (SMA), particularly in those with…
A combination of a ready-to-use SMN protein and an experimental treatment targeting the SMN2 gene, which works in a similar way…
Treatment with the approved gene therapy Zolgensma was found to ease breathing difficulties in children with spinal muscle atrophy…
Gene therapy that selectively delivers the SMN1 gene to the liver reduced alterations related to spinal muscular atrophy (SMA) in…
Some 70% of children with spinal muscle atrophy (SMA) type 1 who underwent treatment with Spinraza alone or…
The use of advanced genetic testing techniques helped clinicians in China to identify novel mutations in…
Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first…