Positive interim data from an ongoing Phase 1 trial in healthy volunteers supports the continued development of Scholar Rock’s…
Articles by Alice Melão
ArcherDX is expanding its product portfolio with newborn and screening tests to identify carriers of genetic variants linked to different…
Administration of Spinraza (nusinersen) directly into the spinal canal is feasible and well-tolerated by adults with spinal…
Novartis Pharmaceuticals‘ investigational compound BVS857 significantly improved muscle volume in patients with X-linked type spinal muscular atrophy (SMA) over a short…
A population-based study conducted in the state of New York demonstrates the feasibility and benefits of newborn screening for spinal…
Targeted therapies or dietary supplements that work to improve the metabolism of certain molecules in muscle cells may slow the…
A working group led by Cure SMA, and comprising 15 experts on spinal muscular atrophy (SMA), has developed new guidelines…
Olesoxime (TRO19622) stabilizes the movement function of patients with Type 2 or Type 3 spinal muscular atrophy…
Levels of a male hormone produced by the adrenal glands could provide useful information on the severity of spinal and…
Using an advanced imaging technique and complex computational algorithms, researchers have created the first detailed, 3D imaging of a group…