Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

Scientists report results from a promising new approach to treat the rare neurodegenerative disorder. Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before ...

On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Then, a ...

Exercise can make a significant difference for patients with SMA by working alongside other medical advancements to improve ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Gyroscope Therapeutics and its gene therapy for the sight-robbing disease ... well as Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy. It's worth noting also that Novartis already ...

although the one-shot therapy is likely to cost in the millions of euros in common with other approved gene therapies such as Novartis' Zolgensma for spinal muscular atrophy at around €2 million.

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...