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 evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...

An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...

Shane Burcaw posted rare photos of himself outside of his own wheelchair, opening up about his courageous decision to do so ...

Neurofilament light (NFL) is a powerful and sensitive biomarker for tracking neuronal health and disease progression.

There is a catch. If the private equity firms can boost sales of bluebird’s three gene therapies to $600 million—over any ...

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 ...

Twilio , the customer engagement platform that drives real-time, personalized experiences for today's leading brands, today announced that Chief Executive Officer Khozema Shipchandler will participate ...

Establishment Labs Holdings Inc. , a global medical technology company dedicated to improving women's health and wellness, principally in breast aesthetics and reconstruction, today announced its ...

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 ...

Researchers at the University of Pittsburgh say a new minimally invasive intervention can target the root cause of neural function loss.

A mother was given SMA treatment Evrysdi during pregnancy, and her child with SMA, now 2.5, has not shown any signs of the ...