Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...
NVS reports new safety and efficacy data from the phase III program for its investigational OAV101 IT gene therapy for SMA ...
Detailed price information for Pacific Biosciences (PACB-Q) from The Globe and Mail including charting and trades.
A child with a rare genetic disorder — spinal muscular atrophy type I — was treated prenatally for the first time. Courtesy of Jakob Owens via Unsplash During an amniocentesis test, a long, thin ...
Monday’s label expansion also continues Roche’s regulatory run in recent months. Last month, for instance, the company’s SMN2 splicing modifier Evrysdi became the first FDA-approved tablet for spinal ...
The SALSA MLPA Probemix P060 SMA Carrier is designed to detect copy number variations in exons 7 and 8 of the SMN1 gene, while the SALSA MLPA Probemix P021 SMA detects copy number variations in SMN1, ...
This work presents an important mouse model for a liver-specific depletion of the Survival Motor Neuron (SMN) protein, where the liver retains 30% of functional full-length SMN protein. The authors ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback