A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

The newly licensed intrathecal gene therapy expands life-changing treatment options for children and adults affected by ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

Spinal muscular atrophy (SMA) and spina bifida are distinct conditions that affect the nerves that allow for voluntary motor control. Share on Pinterest press coverage photography/Getty Images SMA is ...

This image from video provided by UPMC and University of Pittsburgh Health Sciences shows Doug McCullough, who has spinal muscular atrophy, during tests of experimental spinal cord stimulation to ...

People with spinal muscle atrophy (SMA), an inherited neuromuscular disease, usually experience muscle weakness that impacts movement. New research suggests that electrical spinal cord stimulation ...

Novartis’ ITVISMA, approved a month ago by the US FDA, used at Sheikh Khalifa Medical City under the supervision of the ...

The UAE-based Medcare Women and Children Hospital has successfully administered a pioneering intrathecal gene therapy for ...

NBS programs are fairly new in the U.S., so not much is known about variations among them or about provider practice patterns for newborns with SMA. In a survey of providers and state NBS programs, ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. So most recently, there has been the ...

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...

Abu Dhabi marks landmark achievement in becoming the first in the world to deliver ITVISMA (onasemnogene abeparvovec) for the ...