PTC Therapeutics, Inc. announced that their collaborative program with Roche and the SMA Foundation (SMAF) for Spinal Muscular Atrophy…
Articles by Patricia Inacio, PhD
A study entitled “Patient with spinal muscular atrophy with respiratory distress type 1 presenting initially with hypertonia” reports…
In the study entitled “Observational study of spinal muscular atrophy type I and implications for clinical…
In a recent study published in the Molecular Therapy journal, the authors report an improved method to deliver the…
A study entitled “Myogenic program dysregulation is contributory to disease pathogenesis in spinal muscular atrophy” showed that…
A study entitled “A simple and precise diagnostic method for spinal muscular atrophy using a quantitative…
A study entitled “Label-free proteomics identifies Calreticulin and GRP75/Mortalin as peripherally accessible protein biomarkers for spinal muscular atrophy”…
New insights into SMA are shedding light on the genetics that underscore the development of the disease. In a…
A recent study entitled “Analysis of the C9orf72 gene in spinal muscular atrophy patients” published in…
A recent study entitled “SMN regulates axonal local translation via miR-183/mTOR pathway” and published in…