7 April 2017 - No parents should have to watch their child die, yet my former colleague “Will” and his wife “Mary” watched powerless as two of their children succumbed to spinal muscular atrophy. 

“Isaac” and “Lizzy” were never able to sit, talk, or eat on their own, and each passed away as toddlers.

SMA is the most common genetic cause of infant mortality, affecting 1 in 11,000 newborns yearly. Infants with spinal muscular atrophy (SMA) 1, the most common and severe form, develop progressive paralysis before six months. Most die before their second birthday. The genetic cause of SMA, a mutation in the spinal motor neuron 1 gene was discovered 20 years ago, but despite intensive research, no effective therapy was found — until now. 

In December, the U.S. FDA announced its approval of nusinersen (Spinraza), an effective SMA treatment licensed to Biogen by Ionis Pharmaceuticals.

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