New Study Seeks To Improve Performance of Spinal Muscular Atrophy Type I Clinical Trials
In the study entitled “Observational study of spinal muscular atrophy type I and implications for clinical…
Articles by Patricia Inacio, PhD
Virus-Delivered AAV9 SMA Therapy Significantly Improves Outcomes in Animal Models
In a recent study published in the Molecular Therapy journal, the authors report an improved method to deliver the…
Researchers Discover Myogenic Program Dysregulation Contributes to Spinal Muscular Atrophy Disease Pathogenesis
A study entitled “Myogenic program dysregulation is contributory to disease pathogenesis in spinal muscular atrophy” showed that…
High-Resolution CE-SSCP Offers Fast, Accurate Spinal Muscular Atrophy Test
A study entitled “A simple and precise diagnostic method for spinal muscular atrophy using a quantitative…
Key Spinal Muscular Atrophy Biomarkers Discovered
A study entitled “Label-free proteomics identifies Calreticulin and GRP75/Mortalin as peripherally accessible protein biomarkers for spinal muscular atrophy”…
Hypomyelination with Spinal Muscular Atrophy Cerebellar Hypoplasia Caused By EXOSC8 mutations
New insights into SMA are shedding light on the genetics that underscore the development of the disease. In a…
C9orf72 Gene Role Analyzed in Spinal Muscular Atrophy
A recent study entitled “Analysis of the C9orf72 gene in spinal muscular atrophy patients” published in…
Newly-Identified miR-183/mTOR Pathway Contributes to SMA
A recent study entitled “SMN regulates axonal local translation via miR-183/mTOR pathway” and published in…
Therapy That Corrects SMn2 Gene in Spinal Muscular Atrophy Shows Promise
Viable treatments for SMA remain elusive. However, researchers have recently made a series of impressive discoveries in treating the…
Two Recent SMA Studies on Drosophila, Childhood Spinal Muscular Atrophy Could Spur Future Drug Development
Two recent research projects involving studies into Spinal Muscular Atrophy are shedding new light on the deadly condition, which…