Phase 2 Trial Planned for SRK-015 in SMA Patients After Positive Phase 1 Data
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 Enters Newborn Screening Market with Baby Genes Acquisition
ArcherDX is expanding its product portfolio with newborn and screening tests to identify carriers of genetic variants linked to different…
Intrathecal Dosing of Spinraza Is Safe in Adults with SMA Type 2 or 3, Study Shows
Administration of Spinraza (nusinersen) directly into the spinal canal is feasible and well-tolerated by adults with spinal…
BVS857 Clinical Trial Yields Mixed Results as Treatment for Kennedy’s Disease
Novartis Pharmaceuticals‘ investigational compound BVS857 significantly improved muscle volume in patients with X-linked type spinal muscular atrophy (SMA) over a short…
NY Study Shows Feasibility and Benefits of SMA Newborn Screening
A population-based study conducted in the state of New York demonstrates the feasibility and benefits of newborn screening for spinal…
Targeting Essential Amino Acids Seen to Ease Muscular Symptoms of SMA in Mouse Study
Targeted therapies or dietary supplements that work to improve the metabolism of certain molecules in muscle cells may slow the…
New Guidelines Help Define Best Treatment Timeline for SMA Infants
A working group led by Cure SMA, and comprising 15 experts on spinal muscular atrophy (SMA), has developed new guidelines…
#AAN2018 – Olesoxime Stabilizes SMA Patients’ Long-term Movement Function, Extension Study Shows
Olesoxime (TRO19622) stabilizes the movement function of patients with Type 2 or Type 3 spinal muscular atrophy…
Levels of a Male Hormone Could Help Predict SBMA Severity, Study Reports
Levels of a male hormone produced by the adrenal glands could provide useful information on the severity of spinal and…
3D Imaging of Cell’s Transport System May Shed Light on SMA, Other Disorders
Using an advanced imaging technique and complex computational algorithms, researchers have created the first detailed, 3D imaging of a group…