Boy, 5, Born with Rare Genetic Disorder Walks by Himself After Receiving World’s Most Expensive Drug

A 5-year-old boy who was born with a rare genetic condition is now able to walk by himself, his mother has said, after ...

My son was given world's most expensive gene therapy drug - now he can walk

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

What you should know about spinal muscular atrophy

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 ...
The Chosun Ilbo on MSN

British boy walks after world's costliest gene therapy

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, ...
The Chosun Ilbo on MSN

Gene therapy miracle: British boy walks, swims

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 ...
The Caledonian-Record

UAE’s Medcare Treats First International SMA Patient With Revolutionary Intrathecal Gene Therapy

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

Real-world data validates gene therapy for spinal muscular atrophy

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 ...
New Atlas

Infant's genetic muscle disorder improved by treating pregnant mother

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 ...
Arabian Business

Abu Dhabi uses breakthrough gene therapy treatment for spinal muscular atrophy

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

Gene therapy slows progression of spinal muscular atrophy type 2

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an ...
Arabian Business

UAE expands access to gene therapy for spinal muscular atrophy patients

Regulator approves Itvisma for adults and children aged two and above, positioning the UAE among the first countries globally to authorise the treatment for a wider age group ...
Medscape

Gene Therapy Tops Nusinersen in 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 ...