Live Science on MSN14h
Deadly motor-neuron disease treated in the womb in world 1st
An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...
Groundbreaking drug treats rare genetic disorder in the womb for first time ever
The revolutionary drug, called risdiplam, was taken by the mother during her 32nd week of pregnancy and is now part of the ...
News Medical on MSN15h
LUC7 proteins play crucial role in efficient RNA splicing
RNA splicing is a cellular process that is critical for gene expression. After genes are copied from DNA into messenger RNA, portions of the RNA that don't code for proteins, called introns, are cut ...
SMA15h
Child doing well after mother had SMA treatment while pregnant
A mother was given SMA treatment Evrysdi during pregnancy, and her child with SMA, now 2.5, has not shown any signs of the ...
AZoLifeSciences on MSN4h
LUC7 Proteins Play Key Role in RNA Splicing Control
RNA splicing is a cellular process that is critical for gene expression. After genes are copied from DNA into messenger RNA, portions of the RNA that don't code for proteins, called introns, are cut ...
Neurology Advisor17h
FDA Approves Tablet Form of Evrysdi for Spinal Muscular Atrophy
The US Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for people living with spinal muscular atrophy (SMA).
The American Journal of Managed Care16h
Redefining Functional Measures in SMA: Insights From Clinical Research
The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...