The scientists at the University of California, San Francisco, and Johns Hopkins University in Baltimore injected an ...

Evidence is mounting that clinicians can treat serious genetic disorders prenatally by injecting medicine into the amniotic ...

Injecting medicine into the amniotic fluid staves off progression of spinal muscular atrophy in utero.Evidence is mounting ...

Ridhay’s parents received the devastating diagnosis just months ago, and they’ve since been living each day with a renewed ...

Motor improvements in adults with spinal muscular atrophy (SMA ... preserved functionally connected motor neurons without increasing overall motor neuron survival.” Spinraza also restored the nerve ...

Spinal muscular atrophy (SMA) is a progressive motor neuron disorder. While there is a spectrum of ... This has resulted in major motor and survival benefits.5–7 In the past 7 years, disease-modifying ...