Experimental SMA gene therapy helps with head control, sitting up
EXG001-307, an investigational nerve cell-targeting gene therapy being developed by Exegenesis Bio, was well tolerated and helped a small…
Articles by Margarida Maia, PhD
Woman with SMA type 2 gives birth despite breathing problems
A young woman with spinal muscular atrophy (SMA) type 2 gave birth to a premature baby boy…
2D model may speed treatments for SMA, other neuromuscular diseases
Researchers have developed a two-dimensional (2D) model that mimics the crosstalk between nerve and muscle cells. They intend to use…
Younger children on Spinraza made more motor function gains: Study
Being on Spinraza (nusinersen) for up to three years results in a gain in motor function for children with…
Study: Kidney stones may be more common in SMA type 1 children
Children with spinal muscular atrophy (SMA) type 1 may be at an increased risk of developing kidney stones, suggesting…
SMA varies with 4 SMN2 gene copies, gets worse with age
How spinal muscular atrophy (SMA) manifests among people who carry four copies of the SMN2 gene varies, ranging from…
Dosing begins in Phase 2 study of NMD670 for SMA type 3
A first patient has been dosed in a Phase 2 clinical trial evaluating the safety and efficacy of NMD670, an…
Phase 3 trial of apitegromab in SMA type 2 and 3 patients underway
Scholar Rock announced that a Phase 3 clinical trial of its experimental muscle-targeting therapy apitegromab has finished…
MDA grants scientist $300K for research into role of SMN protein
The Muscular Dystrophy Association (MDA) has awarded one of 37 new research grants to a professor studying the role of…
Enrollment complete in Phase 3 trial of taldefgrobep alfa for SMA
Biohaven Pharmaceuticals has completed the enrollment of children and young adults with spinal muscular atrophy (SMA) in a…