The blood creatinine-to-cystatin-c ratio (CCR), an indicator of muscle mass and strength, may be a biomarker of treatment response in…
Articles by Andrea Lobo
The strength of nerve signals to muscles may serve as a marker of disease severity, and as a means to…
The case of a 51-year-old man ultimately diagnosed with spinal muscular atrophy (SMA) type 4 is believed to mark the…
Higher levels of the glial fibrillary acidic protein (GFAP) in the fluid around the brain and spinal cord are associated…
A small molecule known as 10H-phenothiazine (PTZ) reduced the loss of motor neurons, the nerve cells lost in spinal…
Mifepristone, a glucocorticoid blocker, increased survival and muscle size in a mouse model of spinal muscular atrophy (SMA), according…
Treatment with Zolgensma (onasemnogene abeparvovec-xioi) improved motor function in children with spinal muscular atrophy (SMA) who needed tracheostomy,…
The specialty pharmacy Orsini was selected as a partner to provide Itvisma (onasemnogene abeparvovec-brve), the newly approved gene therapy…
A high-dose regimen of nusinersen — the active ingredient in Spinraza — has been recommended for approval to treat…
Blocking a small RNA molecule called miR-140-3b can improve motor function in a mouse model of spinal muscular atrophy…