Preclinical study finds antidepressant may boost SMN
A preclinical study found that blocking the SMPD1 enzyme with available drugs such as the antidepressant clomipramine increases the levels…
Articles by Margarida Maia, PhD
PROTAC therapy shows promise for SBMA, study finds
ARD-1676, a medication that works by tagging toxic proteins for destruction in cells, promotes clearance of the androgen receptor (AR)…
Spinraza can protect adults with SMA from breathing decline
For adults with spinal muscular atrophy (SMA), a genetic disease that progressively weakens breathing muscles, the approved treatment…
FDA denies approval for higher dose of nusinersen for SMA
The U.S. Food and Drug Administration (FDA) has declined to approve a higher dose of nusinersen — the medication in…
Drug combination improves symptoms in SMA mouse model
An experimental oral combination of two therapies eased motor symptoms and prolonged survival in a mouse model of spinal…
Salbutamol may improve upper limb strength in SMA children: Study
The use of salbutamol may safely improve upper limb strength, as well as provide other benefits, in people with…
Spinraza benefits in SMA types 2, 3 seen on muscle MRIs: Study
A quantitative MRI scan, which shows detailed images of muscles, can be used to track changes seen with Spinraza (nusinersen)…
Ride-on cars provide confidence boost for SMA children: Study
Offering children with spinal muscular atrophy (SMA) type 1 the opportunity to move around on their own using motorized,…
School-age SMA children more independent on Spinraza: Study
After Spinraza (nusinersen) treatment for slightly longer than one year, school-aged children with spinal muscular atrophy (SMA) showed…
SMA gene therapy GC101 found safe, effective in animals
Gene therapy GC101 was found to rescue disease manifestations in a mouse model of spinal muscular atrophy (SMA) type…