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

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

A 5-year-old British boy with the rare disease spinal muscular atrophy (SMA) has miraculously started walking four years after receiving the world’s most expensive gene therapy. On the 31st, the BBC ...

A 5-year-old British boy suffering from the rare disease spinal muscular atrophy (SMA) has miraculously begun to walk four years after receiving the "world’s most expensive" gene therapy. On the 31st, ...

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

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 ...

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

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 ...

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 ...