Developed by Novartis, ITVISMA is a one-time therapy for patients aged 2 and older with a confirmed SMN1 gene mutation. The ...

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

Spinal muscular atrophy is a leading genetic cause of infant death worldwide, and is typically caused by loss-of-function mutations in the SMN1 gene, which produces the SMN protein Gene therapy and ...

The parents were both known carriers of SMN1 gene mutations that raised the risk of SMA, and sadly had previously had a child born with the disease who died at 16 months of age. Genetic testing of ...

Spinal muscular atrophy (SMA) is caused by mutations in the SMN1 gene, which encodes survival motor neuron 1, leading to reduced protein expression levels and degeneration of motor neurons in the ...