Posted by Michael Wonder on 01 Mar 2018
Ontario family of toddler with rare disorder seeks funding for expensive drug
27 February 2018 - A family in Windsor, Ontario says they are being forced to choose between spending a fortune for a potentially lifesaving drug, or watching their daughter die.
Nicole Lamont’s two-year-old daughter, Lilah, has a rare condition called spinal muscular atrophy, or SMA. It’s a genetic disease that destroys motor neurons in the spinal cord, damaging children’s ability to move. The disease is progressive, typically leading to paralysis and often death.
Lilah has recently been able to take an injectable drug called Spinraza, or nusinersen, which Health Canada approved for sale in June, 2017. The drug delivers synthetic pieces of DNA to increase a key protein that’s missing in people with SMA and is the first medication to show effectiveness at halting and sometimes reversing SMA.
