Swiss drugmaker Novartis said on Monday its gene therapy helped improve motor function in children with a rare muscle ...

The combination of Spinraza and Zolgensma could improve clinical outcomes in infants with spinal muscular atrophy without ...

Nearly 4 of every 5 infants in Italy born with SMA type 1 since the approval of disease-modifying therapies (DMTs) are alive, ...

The Phase III STEER study met its primary endpoint showing an increase from baseline in HFMSE total score in patients with SMA treated with intrathecal onasemnogene abeparvovec (OAV101 IT) OAV101 IT ...

Roughly 70 per cent of those will have SMA type 1, the most severe of the four clinical subtypes. In Australia, about five to 20 infants with SMA type 1 die each year. The disease is transmitted ...

An 11-month-old baby with spinal muscular atrophy (SMA) needs a Rs 14.2 crore life-saving gene therapy. The Supreme Court ...

Parents should be aware of the common symptoms of Spinal Muscular Atrophy (SMA) that can sometimes be mistaken for other conditions. Infants with SMA may exhibit muscle weakness, especially in the ...

Baby Markuss, who has Type 1 spinal muscular atrophy (SMA), was treated at Sheffield Children's Hospital. The drug has a list price of £1.79m per dose and was made available on the NHS after a ...

to cover the cost of their baby's life-saving treatment. Nine-month-old Pia, who has spinal muscular atrophy (SMA) and lives in Antwerp, requires the drug Zolgensma, which has not been approved ...