Three people with a muscle-destroying disease destined to worsen got a little stronger when an implanted device zapped their ...

Novartis has shared positive top-line results from a late-stage study of its investigational gene therapy in patients aged two to less than 18 years with the rare neuromuscular disease spinal muscular ...

How a tiny spinal implant could help those with muscle-wasting disease - Three people with a muscle-destroying disease got ...

In a small human trial in SMA patients neurostimulation sessions improved motoneuron function, reduced fatigue, and improved leg muscle strength and walking.

This success could broaden the eligible patient population for the gene transfer therapy, according to GlobalData. The study involved paediatric patients aged 2–17 with type II spinal muscular atrophy ...

A new drug-free, minimally invasive intervention targets the root cause of progressive loss of neural function in spinal muscle atrophy (SMA), an inherited neuromuscular disease.

Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 eu ...

in treating individuals with spinal muscular atrophy (SMA) Type 2. The randomised, sham-controlled study assessed the efficacy and safety of the gene therapy in treatment-naïve individuals aged ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...

The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...

Seeking urgent Union government intervention to overcome the lack of affordable access to medicines for treating Spinal Muscular Atrophy ... xioi (Zolgensma) a gene therapy for treating children ...