Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.
Spinal muscular atrophy (SMA), a type of motor neurone disease, is one of the deadliest genetic illnesses an infant can be diagnosed with. Caused by a missing or faulty gene called ‘SMN1’, the disease causes muscles to shrink and become weaker, limiting a baby’s ability to sit, walk, crawl, or even hold their head up.
