Posted by Michael Wonder on 05 Nov 2018
$1 million treatment could save our infants from the orphan disease
4 November 2018 - A $1 million wonder drug labelled as a "game changer" for treating the leading genetic cause of infant and toddler deaths.
Spinal muscular atrophy (SMA) was a degenerative disease that robbed muscles of their ability to function. Babies with SMA type 1 often cannot roll over, sit up, swallow, or reach out for a toy.
The disease was thought to affect around 100 New Zealanders.
