Three years of treatment with apitegromab, a muscle-targeting therapy being developed by Scholar Rock, continues to provide clinical…
Articles by Margarida Maia, PhD
A woman with a long history of muscle weakness was found to have spinal muscular atrophy (SMA) in her…
A method that uses laser light to get information from inside the body in a noninvasive way may help monitor…
EXG001-307, an investigational nerve cell-targeting gene therapy being developed by Exegenesis Bio, was well tolerated and helped a small…
A young woman with spinal muscular atrophy (SMA) type 2 gave birth to a premature baby boy…
Researchers have developed a two-dimensional (2D) model that mimics the crosstalk between nerve and muscle cells. They intend to use…
Being on Spinraza (nusinersen) for up to three years results in a gain in motor function for children with…
Children with spinal muscular atrophy (SMA) type 1 may be at an increased risk of developing kidney stones, suggesting…
How spinal muscular atrophy (SMA) manifests among people who carry four copies of the SMN2 gene varies, ranging from…
A first patient has been dosed in a Phase 2 clinical trial evaluating the safety and efficacy of NMD670, an…