Megan Willis' son, Edward, was given the gene therapy, Zolgensma, after being diagnosed with spinal muscular atrophy when he ...

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

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

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular ...

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

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 ...

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy ...

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

Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child showing no ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Topline results from a phase 3 clinical trial showed an ...

The UAE has become the second country worldwide to approve Itvisma, a groundbreaking gene therapy for spinal muscular atrophy ...

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...