Posted by Michael Wonder on 15 Jan 2018
Parents of kids with rare neuromuscular disorder want funding for expensive drug
15 January 2018 - Parents whose children are battling a genetic disease called spinal muscular atrophy are calling on the provinces to fund a drug that's considered a life-changer, but also one of the most expensive medications in the world.
The drug is called nusinersen, sold under the name Spinraza, and is the only approved treatment for spinal muscular atrophy, or SMA. SMA is a genetic disease that destroys motor neurons in the spinal cord, damaging children’s ability to move. The disease is progressive, leading to paralysis in many, and often death in the most severe forms of the condition.
Spinraza is the first medication shown to be effective at halting and sometimes reversing some of the effects of SMA. The drug is injected into the spine and works by delivering pieces of synthetic DNA to increase a key protein that’s missing in people with SMA.
